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Company Announces Regulatory Filing for Nemolizumab for Two Indications
On February 14, 2024, Galderma announced that .
A first-in-class investigational monoclonal antibody specifically designed to inhibit interleukin (IL) IL-31 signaling, nemolizumab has also been granted FDA Priority Review for prurigo nodularis, according to a press release from the company. The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab for both prurigo nodularis and atopic dermatitis.
The regulatory developments follow data from the phase III OLYMPIA clinical trial program, which evaluated the efficacy and safety of nemolizumab administered subcutaneously every 4 weeks in patients with prurigo nodularis (NCT04501679 and NCT04501666). According to the press release, in OLYMPIA 1 and 2, 58% and 56% of patients, respectively, achieved at least a least four-point reduction in itch intensity as measured by the peak-pruritus numerical rating scale (PP-NRS), compared with 17% and 21% in the placebo groups (P < .0001). At the same time, 26% and 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions on the investigator’s global assessment (IGA) score, compared with 7% and 11% in the placebo groups (P < .0001).
On February 14, 2024, Galderma announced that .
A first-in-class investigational monoclonal antibody specifically designed to inhibit interleukin (IL) IL-31 signaling, nemolizumab has also been granted FDA Priority Review for prurigo nodularis, according to a press release from the company. The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab for both prurigo nodularis and atopic dermatitis.
The regulatory developments follow data from the phase III OLYMPIA clinical trial program, which evaluated the efficacy and safety of nemolizumab administered subcutaneously every 4 weeks in patients with prurigo nodularis (NCT04501679 and NCT04501666). According to the press release, in OLYMPIA 1 and 2, 58% and 56% of patients, respectively, achieved at least a least four-point reduction in itch intensity as measured by the peak-pruritus numerical rating scale (PP-NRS), compared with 17% and 21% in the placebo groups (P < .0001). At the same time, 26% and 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions on the investigator’s global assessment (IGA) score, compared with 7% and 11% in the placebo groups (P < .0001).
On February 14, 2024, Galderma announced that .
A first-in-class investigational monoclonal antibody specifically designed to inhibit interleukin (IL) IL-31 signaling, nemolizumab has also been granted FDA Priority Review for prurigo nodularis, according to a press release from the company. The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab for both prurigo nodularis and atopic dermatitis.
The regulatory developments follow data from the phase III OLYMPIA clinical trial program, which evaluated the efficacy and safety of nemolizumab administered subcutaneously every 4 weeks in patients with prurigo nodularis (NCT04501679 and NCT04501666). According to the press release, in OLYMPIA 1 and 2, 58% and 56% of patients, respectively, achieved at least a least four-point reduction in itch intensity as measured by the peak-pruritus numerical rating scale (PP-NRS), compared with 17% and 21% in the placebo groups (P < .0001). At the same time, 26% and 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions on the investigator’s global assessment (IGA) score, compared with 7% and 11% in the placebo groups (P < .0001).
FDA OKs First Oral Agent for Eosinophilic Esophagitis
Budesonide oral suspension is a corticosteroid indicated for 12 weeks of treatment of EoE in adults and children as young as 11 years.
It will be available in 2-mg/10-mL single-dose stick packs by the end of February.
“Developed specifically for EoE, Eohilia’s novel formulation of budesonide confers thixotropic properties — flowing more freely when shaken and returning to a more viscous state when swallowed,” the company said in a news release. 
“Various formulations of corticosteroids have been used in the past to manage EoE, but in an off-label capacity and using multiple delivery options. With Eohilia, it’s gratifying to now have an FDA-approved treatment specifically formulated for a consistent dose delivery with demonstrated ability to address esophageal inflammation and EoE dysphagia symptoms,” Ikuo Hirano, MD, professor of medicine and director of the Esophageal Center at Northwestern University Feinberg School of Medicine, Chicago, said in the release.
Supporting Data
The FDA approved budesonide oral suspension for EoE based on efficacy and safety data from two multicenter, randomized, double-blind, parallel-group, placebo-controlled 12-week studies.
In study 1, significantly more patients receiving active treatment achieved histologic remission (53.1% vs 1% with placebo). The same was true in study 2, with 38% of patients receiving active treatment achieving histologic remission compared with 2.4% of those receiving placebo.
The absolute change from baseline in the patient-reported Dysphagia Symptom Questionnaire combined score was -10.2 with budesonide vs -6.5 with placebo in Study 1 and -14.5 vs -5.9 in Study 2.
During the last 2 weeks of treatment, more patients receiving budesonide oral suspension experienced no dysphagia or only experienced dysphagia that “got better or cleared up on its own” compared with those receiving placebo, the company said.
The most common adverse reactions seen in the clinical trials of budesonide oral suspension for EoE included respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%), headache (5%), gastroenteritis (3%), throat irritation (3%), adrenal suppression (2%), and erosive esophagitis (2%).
Complete prescribing information is available on the FDA website.
A version of this article appeared on Medscape.com.
Budesonide oral suspension is a corticosteroid indicated for 12 weeks of treatment of EoE in adults and children as young as 11 years.
It will be available in 2-mg/10-mL single-dose stick packs by the end of February.
“Developed specifically for EoE, Eohilia’s novel formulation of budesonide confers thixotropic properties — flowing more freely when shaken and returning to a more viscous state when swallowed,” the company said in a news release.
“Various formulations of corticosteroids have been used in the past to manage EoE, but in an off-label capacity and using multiple delivery options. With Eohilia, it’s gratifying to now have an FDA-approved treatment specifically formulated for a consistent dose delivery with demonstrated ability to address esophageal inflammation and EoE dysphagia symptoms,” Ikuo Hirano, MD, professor of medicine and director of the Esophageal Center at Northwestern University Feinberg School of Medicine, Chicago, said in the release.
Supporting Data
The FDA approved budesonide oral suspension for EoE based on efficacy and safety data from two multicenter, randomized, double-blind, parallel-group, placebo-controlled 12-week studies.
In study 1, significantly more patients receiving active treatment achieved histologic remission (53.1% vs 1% with placebo). The same was true in study 2, with 38% of patients receiving active treatment achieving histologic remission compared with 2.4% of those receiving placebo.
The absolute change from baseline in the patient-reported Dysphagia Symptom Questionnaire combined score was -10.2 with budesonide vs -6.5 with placebo in Study 1 and -14.5 vs -5.9 in Study 2.
During the last 2 weeks of treatment, more patients receiving budesonide oral suspension experienced no dysphagia or only experienced dysphagia that “got better or cleared up on its own” compared with those receiving placebo, the company said.
The most common adverse reactions seen in the clinical trials of budesonide oral suspension for EoE included respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%), headache (5%), gastroenteritis (3%), throat irritation (3%), adrenal suppression (2%), and erosive esophagitis (2%).
Complete prescribing information is available on the FDA website.
A version of this article appeared on Medscape.com.
Budesonide oral suspension is a corticosteroid indicated for 12 weeks of treatment of EoE in adults and children as young as 11 years.
It will be available in 2-mg/10-mL single-dose stick packs by the end of February.
“Developed specifically for EoE, Eohilia’s novel formulation of budesonide confers thixotropic properties — flowing more freely when shaken and returning to a more viscous state when swallowed,” the company said in a news release.
“Various formulations of corticosteroids have been used in the past to manage EoE, but in an off-label capacity and using multiple delivery options. With Eohilia, it’s gratifying to now have an FDA-approved treatment specifically formulated for a consistent dose delivery with demonstrated ability to address esophageal inflammation and EoE dysphagia symptoms,” Ikuo Hirano, MD, professor of medicine and director of the Esophageal Center at Northwestern University Feinberg School of Medicine, Chicago, said in the release.
Supporting Data
The FDA approved budesonide oral suspension for EoE based on efficacy and safety data from two multicenter, randomized, double-blind, parallel-group, placebo-controlled 12-week studies.
In study 1, significantly more patients receiving active treatment achieved histologic remission (53.1% vs 1% with placebo). The same was true in study 2, with 38% of patients receiving active treatment achieving histologic remission compared with 2.4% of those receiving placebo.
The absolute change from baseline in the patient-reported Dysphagia Symptom Questionnaire combined score was -10.2 with budesonide vs -6.5 with placebo in Study 1 and -14.5 vs -5.9 in Study 2.
During the last 2 weeks of treatment, more patients receiving budesonide oral suspension experienced no dysphagia or only experienced dysphagia that “got better or cleared up on its own” compared with those receiving placebo, the company said.
The most common adverse reactions seen in the clinical trials of budesonide oral suspension for EoE included respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%), headache (5%), gastroenteritis (3%), throat irritation (3%), adrenal suppression (2%), and erosive esophagitis (2%).
Complete prescribing information is available on the FDA website.
A version of this article appeared on Medscape.com.
FDA OKs Neuroimaging Tool to Aid Diagnosis of Degenerative Brain Diseases
Research has suggested that neuromelanin is a potential biomarker for neurologic disorders such as Parkinson’s disease.
A recent meta-analysis of 12 neuromelanin MRI studies with 403 patients with Parkinson’s disease and 298 control participants found that neuromelanin MRI had “favorable” diagnostic performance in discriminating patients with Parkinson’s disease from healthy controls.
Until now, there were no FDA-approved devices capable of providing clinicians with analysis of neuromelanin MRI due to a lack of automation and standardization. NM-101 contains algorithms that enable fully automated analysis and the cross-scanner harmonization of neuromelanin MRI scans, the company explains in a news release.
NM-101 is designed to “seamlessly” integrate into existing workflows at hospitals and imaging centers, the company says.
The platform allows clinicians to send neuromelanin MRI images to Terran directly through the hospital picture archiving and communication system and receive results in less than 1 hour.
When interpreted by a neuroradiologist, NM-101 could provide information that may be helpful in determining neuromelanin association as an adjunct to diagnosis.
“We believe this technology could become very important in the clinical workflow of patients with neurological and psychiatric disorders,” Terran Biosciences Founder and CEO Sam Clark, MD, PhD, said in the release.
Neuromelanin MRI has the potential to become “part of the standard of care for the workup of all patients suspected of Parkinson’s and related diseases,” David Sulzer, PhD, professor of neurobiology at Columbia University Vagelos College of Physicians and Surgeons, New York, and co-author of multiple studies using neuromelanin MRI, commented in the news release.
“It’s great to see neuromelanin MRI become more accessible in clinical settings. We hope this opens the door for the adoption of neuromelanin MRI into the clinical workflow for patients with neuropsychiatric disorders,” added Guillermo Horga, MD, PhD, associate professor of psychiatry at Columbia University Vagelos College of Physicians and Surgeons.
Terran Biosciences has an exclusive license to the CNS biomarker software platform and related patents co-owned by Columbia University and Research Foundation for Mental Hygiene. Dr. Horga will receive a portion of the royalties paid to Columbia University for sales of the product.
A version of this article appeared on Medscape.com.
Research has suggested that neuromelanin is a potential biomarker for neurologic disorders such as Parkinson’s disease.
A recent meta-analysis of 12 neuromelanin MRI studies with 403 patients with Parkinson’s disease and 298 control participants found that neuromelanin MRI had “favorable” diagnostic performance in discriminating patients with Parkinson’s disease from healthy controls.
Until now, there were no FDA-approved devices capable of providing clinicians with analysis of neuromelanin MRI due to a lack of automation and standardization. NM-101 contains algorithms that enable fully automated analysis and the cross-scanner harmonization of neuromelanin MRI scans, the company explains in a news release.
NM-101 is designed to “seamlessly” integrate into existing workflows at hospitals and imaging centers, the company says.
The platform allows clinicians to send neuromelanin MRI images to Terran directly through the hospital picture archiving and communication system and receive results in less than 1 hour.
When interpreted by a neuroradiologist, NM-101 could provide information that may be helpful in determining neuromelanin association as an adjunct to diagnosis.
“We believe this technology could become very important in the clinical workflow of patients with neurological and psychiatric disorders,” Terran Biosciences Founder and CEO Sam Clark, MD, PhD, said in the release.
Neuromelanin MRI has the potential to become “part of the standard of care for the workup of all patients suspected of Parkinson’s and related diseases,” David Sulzer, PhD, professor of neurobiology at Columbia University Vagelos College of Physicians and Surgeons, New York, and co-author of multiple studies using neuromelanin MRI, commented in the news release.
“It’s great to see neuromelanin MRI become more accessible in clinical settings. We hope this opens the door for the adoption of neuromelanin MRI into the clinical workflow for patients with neuropsychiatric disorders,” added Guillermo Horga, MD, PhD, associate professor of psychiatry at Columbia University Vagelos College of Physicians and Surgeons.
Terran Biosciences has an exclusive license to the CNS biomarker software platform and related patents co-owned by Columbia University and Research Foundation for Mental Hygiene. Dr. Horga will receive a portion of the royalties paid to Columbia University for sales of the product.
A version of this article appeared on Medscape.com.
Research has suggested that neuromelanin is a potential biomarker for neurologic disorders such as Parkinson’s disease.
A recent meta-analysis of 12 neuromelanin MRI studies with 403 patients with Parkinson’s disease and 298 control participants found that neuromelanin MRI had “favorable” diagnostic performance in discriminating patients with Parkinson’s disease from healthy controls.
Until now, there were no FDA-approved devices capable of providing clinicians with analysis of neuromelanin MRI due to a lack of automation and standardization. NM-101 contains algorithms that enable fully automated analysis and the cross-scanner harmonization of neuromelanin MRI scans, the company explains in a news release.
NM-101 is designed to “seamlessly” integrate into existing workflows at hospitals and imaging centers, the company says.
The platform allows clinicians to send neuromelanin MRI images to Terran directly through the hospital picture archiving and communication system and receive results in less than 1 hour.
When interpreted by a neuroradiologist, NM-101 could provide information that may be helpful in determining neuromelanin association as an adjunct to diagnosis.
“We believe this technology could become very important in the clinical workflow of patients with neurological and psychiatric disorders,” Terran Biosciences Founder and CEO Sam Clark, MD, PhD, said in the release.
Neuromelanin MRI has the potential to become “part of the standard of care for the workup of all patients suspected of Parkinson’s and related diseases,” David Sulzer, PhD, professor of neurobiology at Columbia University Vagelos College of Physicians and Surgeons, New York, and co-author of multiple studies using neuromelanin MRI, commented in the news release.
“It’s great to see neuromelanin MRI become more accessible in clinical settings. We hope this opens the door for the adoption of neuromelanin MRI into the clinical workflow for patients with neuropsychiatric disorders,” added Guillermo Horga, MD, PhD, associate professor of psychiatry at Columbia University Vagelos College of Physicians and Surgeons.
Terran Biosciences has an exclusive license to the CNS biomarker software platform and related patents co-owned by Columbia University and Research Foundation for Mental Hygiene. Dr. Horga will receive a portion of the royalties paid to Columbia University for sales of the product.
A version of this article appeared on Medscape.com.
Top US Oncology Regulator Seeks Changes in Drug Studies
Richard Pazdur, MD, who leads the cancer division at the US Food and Drug Administration (FDA), said there’s a need to simplify the paperwork involved in clinical trials. Before joining the FDA in 1999, Dr. Pazdur participated in and published cancer research. He says the informed consent forms used for studies have grown too elaborate over the years, such that they can intimidate even experts.
“When I read informed consents now in clinical trials, folks, it gives me a headache. Okay, I can’t follow them,” Dr. Pazdur said.
Dr. Pazdur said informed consent forms can be “mind-boggling” these days.
“They’re so damn complicated with so many damn questions being answered,” he said. “So our point is what’s the essential question that you need answered and what’s the quickest way of answering that question with the least amount of data that can be collected?”
Dr. Pazdur made these comments during a joint meeting of the FDA and the European Medicines Agency (EMA).
The meeting was a broad discussion about how to build on the successes seen in treatment of blood cancers in the past two decades. No formal recommendations were introduced or considered at the meeting. Instead, the meeting served as a chance for oncologists and patients to discuss ways to more quickly and efficiently address the key questions in drug research: Do medicines deliver a significant benefit to patients?
Dr. Pazdur also said at the meeting that there needs to be a way to attract more people to enroll in clinical trials.
“When I started in oncology, it was about 5%. When I’m sitting here now, 40 years later, it’s 5%. Basically it hasn’t moved,” he said at the discussion, held on February 1.
Ellin Berman, MD, of Memorial Sloan Kettering Cancer Center in New York, spoke at the meeting about the changes she has witnessed in her career in oncology. Until 2001, there were limited drug options, and physicians tried to get patients to transplant teams as possible. Then the FDA in 2001 approved imatinib to treat patients with chronic myelogenous leukemia (CML) that has the Philadelphia chromosome. That set the stage, Dr. Berman said, for a sea change in treatment of CML.
“The fellows now have no idea what it is like to talk to a CML patient about transplant and the question is which among the treasures we have of drugs do we start people on? And that’s always a conversation,” Dr. Berman said.
She noted that advances in treatment have also let some female patients get pregnant and have children.
“We have at least half a dozen women who bring their kids to clinic. And boy, if that doesn’t bring tears to our eyes, our collective eyes, I don’t know what does,” she said.
Dr. Pazdur also recalled his experience treating patients in the 1970s and 1980s for cancers for which “you had nothing so to speak” in terms of effective treatment.
“So then ask yourself the question, what would their stories be now?” with the many options available, Dr. Pazdur said.
Seeking clinician feedback
To try to improve the development and testing of cancer drugs, the FDA is seeking to get more feedback from clinicians about which questions trials should address, Dr. Pazdur said.
The agency is considering a way to poll clinicians on what their most crucial questions are about the medicines, he said. Better design of trial questions might serve to improve enrollment in studies.
“What we’re thinking of doing is taking the common disease areas and asking clinicians what are the five basic questions that you want answered in the next 5 years,” he said.
He cited PD-1 drugs as a possible example of a class where regulators could consider new approaches. There could be a discussion about the safety data collection for this class of drugs, which has been used by millions of patients.
Dr. Pazdur said he has been discussing these kinds of themes with his European and Japanese counterparts, who also are interested in simplifying clinical trials.
The goal is to have trials better represent real-world experiences rather than “artificial” ones created when patients must meet extensive eligibility requirements. Improved use of emerging technologies could aid in the needed streamlining, Dr. Pazdur said.
“As an oncology community, we have made our lives somewhat too complicated and need to draw back and ask the basic questions,” Dr. Pazdur said.
Richard Pazdur, MD, who leads the cancer division at the US Food and Drug Administration (FDA), said there’s a need to simplify the paperwork involved in clinical trials. Before joining the FDA in 1999, Dr. Pazdur participated in and published cancer research. He says the informed consent forms used for studies have grown too elaborate over the years, such that they can intimidate even experts.
“When I read informed consents now in clinical trials, folks, it gives me a headache. Okay, I can’t follow them,” Dr. Pazdur said.
Dr. Pazdur said informed consent forms can be “mind-boggling” these days.
“They’re so damn complicated with so many damn questions being answered,” he said. “So our point is what’s the essential question that you need answered and what’s the quickest way of answering that question with the least amount of data that can be collected?”
Dr. Pazdur made these comments during a joint meeting of the FDA and the European Medicines Agency (EMA).
The meeting was a broad discussion about how to build on the successes seen in treatment of blood cancers in the past two decades. No formal recommendations were introduced or considered at the meeting. Instead, the meeting served as a chance for oncologists and patients to discuss ways to more quickly and efficiently address the key questions in drug research: Do medicines deliver a significant benefit to patients?
Dr. Pazdur also said at the meeting that there needs to be a way to attract more people to enroll in clinical trials.
“When I started in oncology, it was about 5%. When I’m sitting here now, 40 years later, it’s 5%. Basically it hasn’t moved,” he said at the discussion, held on February 1.
Ellin Berman, MD, of Memorial Sloan Kettering Cancer Center in New York, spoke at the meeting about the changes she has witnessed in her career in oncology. Until 2001, there were limited drug options, and physicians tried to get patients to transplant teams as possible. Then the FDA in 2001 approved imatinib to treat patients with chronic myelogenous leukemia (CML) that has the Philadelphia chromosome. That set the stage, Dr. Berman said, for a sea change in treatment of CML.
“The fellows now have no idea what it is like to talk to a CML patient about transplant and the question is which among the treasures we have of drugs do we start people on? And that’s always a conversation,” Dr. Berman said.
She noted that advances in treatment have also let some female patients get pregnant and have children.
“We have at least half a dozen women who bring their kids to clinic. And boy, if that doesn’t bring tears to our eyes, our collective eyes, I don’t know what does,” she said.
Dr. Pazdur also recalled his experience treating patients in the 1970s and 1980s for cancers for which “you had nothing so to speak” in terms of effective treatment.
“So then ask yourself the question, what would their stories be now?” with the many options available, Dr. Pazdur said.
Seeking clinician feedback
To try to improve the development and testing of cancer drugs, the FDA is seeking to get more feedback from clinicians about which questions trials should address, Dr. Pazdur said.
The agency is considering a way to poll clinicians on what their most crucial questions are about the medicines, he said. Better design of trial questions might serve to improve enrollment in studies.
“What we’re thinking of doing is taking the common disease areas and asking clinicians what are the five basic questions that you want answered in the next 5 years,” he said.
He cited PD-1 drugs as a possible example of a class where regulators could consider new approaches. There could be a discussion about the safety data collection for this class of drugs, which has been used by millions of patients.
Dr. Pazdur said he has been discussing these kinds of themes with his European and Japanese counterparts, who also are interested in simplifying clinical trials.
The goal is to have trials better represent real-world experiences rather than “artificial” ones created when patients must meet extensive eligibility requirements. Improved use of emerging technologies could aid in the needed streamlining, Dr. Pazdur said.
“As an oncology community, we have made our lives somewhat too complicated and need to draw back and ask the basic questions,” Dr. Pazdur said.
Richard Pazdur, MD, who leads the cancer division at the US Food and Drug Administration (FDA), said there’s a need to simplify the paperwork involved in clinical trials. Before joining the FDA in 1999, Dr. Pazdur participated in and published cancer research. He says the informed consent forms used for studies have grown too elaborate over the years, such that they can intimidate even experts.
“When I read informed consents now in clinical trials, folks, it gives me a headache. Okay, I can’t follow them,” Dr. Pazdur said.
Dr. Pazdur said informed consent forms can be “mind-boggling” these days.
“They’re so damn complicated with so many damn questions being answered,” he said. “So our point is what’s the essential question that you need answered and what’s the quickest way of answering that question with the least amount of data that can be collected?”
Dr. Pazdur made these comments during a joint meeting of the FDA and the European Medicines Agency (EMA).
The meeting was a broad discussion about how to build on the successes seen in treatment of blood cancers in the past two decades. No formal recommendations were introduced or considered at the meeting. Instead, the meeting served as a chance for oncologists and patients to discuss ways to more quickly and efficiently address the key questions in drug research: Do medicines deliver a significant benefit to patients?
Dr. Pazdur also said at the meeting that there needs to be a way to attract more people to enroll in clinical trials.
“When I started in oncology, it was about 5%. When I’m sitting here now, 40 years later, it’s 5%. Basically it hasn’t moved,” he said at the discussion, held on February 1.
Ellin Berman, MD, of Memorial Sloan Kettering Cancer Center in New York, spoke at the meeting about the changes she has witnessed in her career in oncology. Until 2001, there were limited drug options, and physicians tried to get patients to transplant teams as possible. Then the FDA in 2001 approved imatinib to treat patients with chronic myelogenous leukemia (CML) that has the Philadelphia chromosome. That set the stage, Dr. Berman said, for a sea change in treatment of CML.
“The fellows now have no idea what it is like to talk to a CML patient about transplant and the question is which among the treasures we have of drugs do we start people on? And that’s always a conversation,” Dr. Berman said.
She noted that advances in treatment have also let some female patients get pregnant and have children.
“We have at least half a dozen women who bring their kids to clinic. And boy, if that doesn’t bring tears to our eyes, our collective eyes, I don’t know what does,” she said.
Dr. Pazdur also recalled his experience treating patients in the 1970s and 1980s for cancers for which “you had nothing so to speak” in terms of effective treatment.
“So then ask yourself the question, what would their stories be now?” with the many options available, Dr. Pazdur said.
Seeking clinician feedback
To try to improve the development and testing of cancer drugs, the FDA is seeking to get more feedback from clinicians about which questions trials should address, Dr. Pazdur said.
The agency is considering a way to poll clinicians on what their most crucial questions are about the medicines, he said. Better design of trial questions might serve to improve enrollment in studies.
“What we’re thinking of doing is taking the common disease areas and asking clinicians what are the five basic questions that you want answered in the next 5 years,” he said.
He cited PD-1 drugs as a possible example of a class where regulators could consider new approaches. There could be a discussion about the safety data collection for this class of drugs, which has been used by millions of patients.
Dr. Pazdur said he has been discussing these kinds of themes with his European and Japanese counterparts, who also are interested in simplifying clinical trials.
The goal is to have trials better represent real-world experiences rather than “artificial” ones created when patients must meet extensive eligibility requirements. Improved use of emerging technologies could aid in the needed streamlining, Dr. Pazdur said.
“As an oncology community, we have made our lives somewhat too complicated and need to draw back and ask the basic questions,” Dr. Pazdur said.
FDA Recommends DEA Move Cannabis From Schedule I to III
Newly released documents show that .
The FDA’s recommendation was contained in a 252-page report that was sent to the US Drug Enforcement Administration (DEA) in August 2023. The report, which Bloomberg News reported on in late August and may have been leaked to that news outlet, was released to Houston attorney Matthew Zorn. He filed suit in September to pressure the FDA to make its recommendation public. The FDA responded days before a court-ordered deadline, said Zorn.
The attorney was not representing any client. “This document belongs in the public sphere,” Zorn told this news organization. “I found it farcical that public policy was being debated on the basis of a document recommendation that literally no one had seen,” he said.
The Bloomberg report ignited debate, but no other advocate, attorney, or news organization had been able to obtain an unredacted version of FDA’s recommendation.
Now that the full report is public, the DEA may be under more pressure to act. However, it is not required to do anything, and there is no set timeline for any action. Still, lawyers expect to quickly see a rule proposing moving cannabis from Schedule I to III.
“I expect it to come fairly soon and the reason I expect that is because the President told the agencies to do this expeditiously,” said Shane Pennington, an attorney with Porter Wright who has worked with Zorn on cases challenging DEA’s scheduling process but was not involved in this suit.
In October 2022, President Joe Biden said that he was asking the Department of Health and Human Services and the US Attorney General “to review expeditiously how marijuana is scheduled under federal law.”
Howard Sklamberg, a lawyer with Arnold & Porter in Washington, DC, said that the Biden directive “certainly made the agencies reconsider” rescheduling cannabis but that it likely was going to happen anyway, given a wealth of supportive information generated since the DEA last rejected a rescheduling petition in 2016.
Mr. Sklamberg told this news organization that he thought a proposed rule would be issued soon, with a final rule issued by mid-summer.
“Agencies generally want to get their important rulemaking done before you get too much into the political season and the potential end of a presidency,” said Mr. Sklamberg, a former FDA deputy commissioner.
Credible Medical Use
The FDA said in its report that cannabis is a low-risk threat to public health and that it poses less potential for misuse than drugs in schedule I or II, such as heroin or cocaine.
Though the evidence showed that some people are using cannabis “in amounts sufficient to create a hazard to their health and to the safety of other individuals and the community evidence also exists showing that the vast majority of individuals who use marijuana are doing so in a manner that does not lead to dangerous outcomes to themselves or others,” the FDA noted.
The agency stated that “the risks to the public health posed by marijuana are low compared to other drugs of abuse (e.g., heroin, cocaine, benzodiazepines), based on an evaluation of various epidemiological databases for [emergency department] visits, hospitalizations, unintentional exposures, and most importantly, for overdose deaths.”
The FDA assessed cannabis’s commonly accepted medical use in seven indications: anorexia, anxiety, epilepsy, inflammatory bowel disease, nausea and vomiting, pain, and posttraumatic stress disorder. It concluded that the strongest evidence existed for anorexia related to a medical condition, nausea and vomiting, and pain.
Of interest, the agency said that when it assessed the harms and benefits, it also used alcohol as a comparator even though it is not a controlled substance. The agency said that it did so because of alcohol’s extensive availability and use, “which is also observed for nonmedical use of marijuana.”
Mr. Sklamberg found that interesting. A majority of adults have consumed cannabis or know someone who has, making it similar to alcohol, he said. And just as with alcohol, “those adults have formed their own conclusions about what marijuana is and what it isn’t,” he said.
“A lot of Americans make their judgment and think schedule I overstates the health risks,” he added.
Opposition in Congress
It is not certain whether cannabis will be rescheduled; after the Bloomberg report in August, Republican members of Congress sent a letter to DEA Administrator Anne Milgram telling her that the agency should not reschedule the drug.
“The recommendation to remove cannabis from the DEA’s list of dangerous Schedule I drugs is not based on science — it’s based on an irresponsible pro-pot agenda,” said Oklahoma Senator James Lankford (R) on X, in September.
The letter contended that there is no accepted medical use for cannabis and that “the known facts about marijuana have not changed since 2016.”
The FDA, however, based its recommendations in part in looking at data from more than 30,000 healthcare providers and six million patients who have used medical marijuana in state programs, largely established since 2016. Congress has directed the agency to evaluate more of that kind of real-world evidence when evaluating products, said Mr. Sklamberg.
He said that the FDA report will be taken seriously: “It’s a thorough and impressive document.”
“It’s not a document that looks like it was just put together by policy people or political people,” Mr. Sklamberg added. “It’s heavily grounded in science and medicine.”
A version of this article appeared on Medscape.com.
Newly released documents show that .
The FDA’s recommendation was contained in a 252-page report that was sent to the US Drug Enforcement Administration (DEA) in August 2023. The report, which Bloomberg News reported on in late August and may have been leaked to that news outlet, was released to Houston attorney Matthew Zorn. He filed suit in September to pressure the FDA to make its recommendation public. The FDA responded days before a court-ordered deadline, said Zorn.
The attorney was not representing any client. “This document belongs in the public sphere,” Zorn told this news organization. “I found it farcical that public policy was being debated on the basis of a document recommendation that literally no one had seen,” he said.
The Bloomberg report ignited debate, but no other advocate, attorney, or news organization had been able to obtain an unredacted version of FDA’s recommendation.
Now that the full report is public, the DEA may be under more pressure to act. However, it is not required to do anything, and there is no set timeline for any action. Still, lawyers expect to quickly see a rule proposing moving cannabis from Schedule I to III.
“I expect it to come fairly soon and the reason I expect that is because the President told the agencies to do this expeditiously,” said Shane Pennington, an attorney with Porter Wright who has worked with Zorn on cases challenging DEA’s scheduling process but was not involved in this suit.
In October 2022, President Joe Biden said that he was asking the Department of Health and Human Services and the US Attorney General “to review expeditiously how marijuana is scheduled under federal law.”
Howard Sklamberg, a lawyer with Arnold & Porter in Washington, DC, said that the Biden directive “certainly made the agencies reconsider” rescheduling cannabis but that it likely was going to happen anyway, given a wealth of supportive information generated since the DEA last rejected a rescheduling petition in 2016.
Mr. Sklamberg told this news organization that he thought a proposed rule would be issued soon, with a final rule issued by mid-summer.
“Agencies generally want to get their important rulemaking done before you get too much into the political season and the potential end of a presidency,” said Mr. Sklamberg, a former FDA deputy commissioner.
Credible Medical Use
The FDA said in its report that cannabis is a low-risk threat to public health and that it poses less potential for misuse than drugs in schedule I or II, such as heroin or cocaine.
Though the evidence showed that some people are using cannabis “in amounts sufficient to create a hazard to their health and to the safety of other individuals and the community evidence also exists showing that the vast majority of individuals who use marijuana are doing so in a manner that does not lead to dangerous outcomes to themselves or others,” the FDA noted.
The agency stated that “the risks to the public health posed by marijuana are low compared to other drugs of abuse (e.g., heroin, cocaine, benzodiazepines), based on an evaluation of various epidemiological databases for [emergency department] visits, hospitalizations, unintentional exposures, and most importantly, for overdose deaths.”
The FDA assessed cannabis’s commonly accepted medical use in seven indications: anorexia, anxiety, epilepsy, inflammatory bowel disease, nausea and vomiting, pain, and posttraumatic stress disorder. It concluded that the strongest evidence existed for anorexia related to a medical condition, nausea and vomiting, and pain.
Of interest, the agency said that when it assessed the harms and benefits, it also used alcohol as a comparator even though it is not a controlled substance. The agency said that it did so because of alcohol’s extensive availability and use, “which is also observed for nonmedical use of marijuana.”
Mr. Sklamberg found that interesting. A majority of adults have consumed cannabis or know someone who has, making it similar to alcohol, he said. And just as with alcohol, “those adults have formed their own conclusions about what marijuana is and what it isn’t,” he said.
“A lot of Americans make their judgment and think schedule I overstates the health risks,” he added.
Opposition in Congress
It is not certain whether cannabis will be rescheduled; after the Bloomberg report in August, Republican members of Congress sent a letter to DEA Administrator Anne Milgram telling her that the agency should not reschedule the drug.
“The recommendation to remove cannabis from the DEA’s list of dangerous Schedule I drugs is not based on science — it’s based on an irresponsible pro-pot agenda,” said Oklahoma Senator James Lankford (R) on X, in September.
The letter contended that there is no accepted medical use for cannabis and that “the known facts about marijuana have not changed since 2016.”
The FDA, however, based its recommendations in part in looking at data from more than 30,000 healthcare providers and six million patients who have used medical marijuana in state programs, largely established since 2016. Congress has directed the agency to evaluate more of that kind of real-world evidence when evaluating products, said Mr. Sklamberg.
He said that the FDA report will be taken seriously: “It’s a thorough and impressive document.”
“It’s not a document that looks like it was just put together by policy people or political people,” Mr. Sklamberg added. “It’s heavily grounded in science and medicine.”
A version of this article appeared on Medscape.com.
Newly released documents show that .
The FDA’s recommendation was contained in a 252-page report that was sent to the US Drug Enforcement Administration (DEA) in August 2023. The report, which Bloomberg News reported on in late August and may have been leaked to that news outlet, was released to Houston attorney Matthew Zorn. He filed suit in September to pressure the FDA to make its recommendation public. The FDA responded days before a court-ordered deadline, said Zorn.
The attorney was not representing any client. “This document belongs in the public sphere,” Zorn told this news organization. “I found it farcical that public policy was being debated on the basis of a document recommendation that literally no one had seen,” he said.
The Bloomberg report ignited debate, but no other advocate, attorney, or news organization had been able to obtain an unredacted version of FDA’s recommendation.
Now that the full report is public, the DEA may be under more pressure to act. However, it is not required to do anything, and there is no set timeline for any action. Still, lawyers expect to quickly see a rule proposing moving cannabis from Schedule I to III.
“I expect it to come fairly soon and the reason I expect that is because the President told the agencies to do this expeditiously,” said Shane Pennington, an attorney with Porter Wright who has worked with Zorn on cases challenging DEA’s scheduling process but was not involved in this suit.
In October 2022, President Joe Biden said that he was asking the Department of Health and Human Services and the US Attorney General “to review expeditiously how marijuana is scheduled under federal law.”
Howard Sklamberg, a lawyer with Arnold & Porter in Washington, DC, said that the Biden directive “certainly made the agencies reconsider” rescheduling cannabis but that it likely was going to happen anyway, given a wealth of supportive information generated since the DEA last rejected a rescheduling petition in 2016.
Mr. Sklamberg told this news organization that he thought a proposed rule would be issued soon, with a final rule issued by mid-summer.
“Agencies generally want to get their important rulemaking done before you get too much into the political season and the potential end of a presidency,” said Mr. Sklamberg, a former FDA deputy commissioner.
Credible Medical Use
The FDA said in its report that cannabis is a low-risk threat to public health and that it poses less potential for misuse than drugs in schedule I or II, such as heroin or cocaine.
Though the evidence showed that some people are using cannabis “in amounts sufficient to create a hazard to their health and to the safety of other individuals and the community evidence also exists showing that the vast majority of individuals who use marijuana are doing so in a manner that does not lead to dangerous outcomes to themselves or others,” the FDA noted.
The agency stated that “the risks to the public health posed by marijuana are low compared to other drugs of abuse (e.g., heroin, cocaine, benzodiazepines), based on an evaluation of various epidemiological databases for [emergency department] visits, hospitalizations, unintentional exposures, and most importantly, for overdose deaths.”
The FDA assessed cannabis’s commonly accepted medical use in seven indications: anorexia, anxiety, epilepsy, inflammatory bowel disease, nausea and vomiting, pain, and posttraumatic stress disorder. It concluded that the strongest evidence existed for anorexia related to a medical condition, nausea and vomiting, and pain.
Of interest, the agency said that when it assessed the harms and benefits, it also used alcohol as a comparator even though it is not a controlled substance. The agency said that it did so because of alcohol’s extensive availability and use, “which is also observed for nonmedical use of marijuana.”
Mr. Sklamberg found that interesting. A majority of adults have consumed cannabis or know someone who has, making it similar to alcohol, he said. And just as with alcohol, “those adults have formed their own conclusions about what marijuana is and what it isn’t,” he said.
“A lot of Americans make their judgment and think schedule I overstates the health risks,” he added.
Opposition in Congress
It is not certain whether cannabis will be rescheduled; after the Bloomberg report in August, Republican members of Congress sent a letter to DEA Administrator Anne Milgram telling her that the agency should not reschedule the drug.
“The recommendation to remove cannabis from the DEA’s list of dangerous Schedule I drugs is not based on science — it’s based on an irresponsible pro-pot agenda,” said Oklahoma Senator James Lankford (R) on X, in September.
The letter contended that there is no accepted medical use for cannabis and that “the known facts about marijuana have not changed since 2016.”
The FDA, however, based its recommendations in part in looking at data from more than 30,000 healthcare providers and six million patients who have used medical marijuana in state programs, largely established since 2016. Congress has directed the agency to evaluate more of that kind of real-world evidence when evaluating products, said Mr. Sklamberg.
He said that the FDA report will be taken seriously: “It’s a thorough and impressive document.”
“It’s not a document that looks like it was just put together by policy people or political people,” Mr. Sklamberg added. “It’s heavily grounded in science and medicine.”
A version of this article appeared on Medscape.com.
CRISPR-Based Gene Therapy Earns Beta Thalassemia Approval
The approval, which comes more than 2 months ahead of a target action date of March 30, marks the second for the landmark therapy. The FDA greenlit the CRISPR gene therapy to treat sickle cell disease last December.
The autologous, ex vivo, CRISPR/Cas9 gene-edited therapy from Vertex and CRISPR Therapeutics is the first to use the gene-editing tool CRISPR.
The transfusion-dependent beta thalassemia approval is based on data from pivotal studies showing “consistent and durable response to treatment” in 52 patients who received an infusion and followed for up to 4 years. Treatment conferred transfusion independence in patients with transfusion-dependent beta thalassemia, according to a press release from Vertex late last year.
Vertex noted in a new press statement that expanded approval means about 1000 patients aged 12 years or older will be eligible for the one-time treatment for this indication.
Exa-cel requires administration at authorized treatment centers experienced in stem cell transplantation.
The therapy, which has a list price of $2.2 million in the United States, should be available initially at nine authorized treatment centers early this year, with more to come, according to Vertex.
A version of this article appeared on Medscape.com.
The approval, which comes more than 2 months ahead of a target action date of March 30, marks the second for the landmark therapy. The FDA greenlit the CRISPR gene therapy to treat sickle cell disease last December.
The autologous, ex vivo, CRISPR/Cas9 gene-edited therapy from Vertex and CRISPR Therapeutics is the first to use the gene-editing tool CRISPR.
The transfusion-dependent beta thalassemia approval is based on data from pivotal studies showing “consistent and durable response to treatment” in 52 patients who received an infusion and followed for up to 4 years. Treatment conferred transfusion independence in patients with transfusion-dependent beta thalassemia, according to a press release from Vertex late last year.
Vertex noted in a new press statement that expanded approval means about 1000 patients aged 12 years or older will be eligible for the one-time treatment for this indication.
Exa-cel requires administration at authorized treatment centers experienced in stem cell transplantation.
The therapy, which has a list price of $2.2 million in the United States, should be available initially at nine authorized treatment centers early this year, with more to come, according to Vertex.
A version of this article appeared on Medscape.com.
The approval, which comes more than 2 months ahead of a target action date of March 30, marks the second for the landmark therapy. The FDA greenlit the CRISPR gene therapy to treat sickle cell disease last December.
The autologous, ex vivo, CRISPR/Cas9 gene-edited therapy from Vertex and CRISPR Therapeutics is the first to use the gene-editing tool CRISPR.
The transfusion-dependent beta thalassemia approval is based on data from pivotal studies showing “consistent and durable response to treatment” in 52 patients who received an infusion and followed for up to 4 years. Treatment conferred transfusion independence in patients with transfusion-dependent beta thalassemia, according to a press release from Vertex late last year.
Vertex noted in a new press statement that expanded approval means about 1000 patients aged 12 years or older will be eligible for the one-time treatment for this indication.
Exa-cel requires administration at authorized treatment centers experienced in stem cell transplantation.
The therapy, which has a list price of $2.2 million in the United States, should be available initially at nine authorized treatment centers early this year, with more to come, according to Vertex.
A version of this article appeared on Medscape.com.
FDA Clears AI-Powered Device for Noninvasive Skin Cancer Testing
The handheld wireless tool, which was developed by Miami-based DermaSensor Inc., operates on battery power, uses spectroscopy and algorithms to evaluate skin lesions for potential cancer in a matter of seconds, and is intended for use by primary care physicians. After the device completes the scan of a lesion, a result of “investigate further” (positive result) suggests further evaluation through a referral to a dermatologist, while “monitor” (negative result) suggests that there is no immediate need for a referral to a dermatologist.
In a pivotal trial of the device that evaluated 224 high risk lesions at 18 primary care study sites in the United States and 4 in Australia, the device had an overall sensitivity of 95.5% for detecting malignancy.
In a more recent validation study funded by DermaSensor, investigators tested 333 lesions at four U.S. dermatology offices and found that the overall device sensitivity was 97.04%, with subgroup sensitivity of 96.67% for melanoma, 97.22% for basal cell carcinoma, and 97.01% for squamous cell carcinoma. Overall specificity of the device was 26.22%.
The study authors, led by Tallahassee, Fla.–based dermatologist Armand B. Cognetta Jr., MD, concluded that DermaSensor’s rapid clinical analysis of lesions “allows for its easy integration into clinical practice infrastructures. Proper use of this device may aid in the reduction of morbidity and mortality associated with skin cancer through expedited and enhanced detection and intervention.”
According to marketing material from the DermaSensor website, the device’s AI algorithm was developed and validated with more than 20,000 scans, composed of more than 4,000 benign and malignant lesions. In a statement about the clearance, the FDA emphasized that the device “should not be used as the sole diagnostic criterion nor to confirm a diagnosis of skin cancer.” The agency is requiring that the manufacturer “conduct additional post-market clinical validation performance testing of the DermaSensor device in patients from demographic groups representative of the U.S. population, including populations who had limited representation of melanomas in the premarket studies, due to their having a relatively low incidence of the disease.”
According to a spokesperson for DermaSensor, pricing for the device is based on a subscription model: $199 per month for five patients or $399 per month for unlimited use. DermaSensor is currently commercially available in Europe and Australia.
Asked to comment, Vishal A. Patel, MD, director of cutaneous oncology at the George Washington Cancer Center, Washington, said that the FDA clearance of DermaSensor highlights the growing appreciation of AI-driven diagnostic support for primary care providers and dermatologists. "Skin cancers are a growing epidemic in the US and the ability to accurately identify potential suspicious lesions without immediately reaching for the scalpel is invaluable," Patel told this news organization. He was not involved with DermSensor studies.
"Furthermore, this tool can help address the shortage of dermatologists and long wait times by helping primary care providers accurately risk-stratify patients and identify those who need to be seen immediately for potential biopsy and expert care," he added. "However, just like with any new technology, we must use caution to not overutilize this tool," which he said, could "lead to overdiagnosis and overtreatment of early or innocuous lesions that are better managed with empiric field treatments."
Dr. Cognetta was a paid investigator for the study.
Dr. Patel disclosed that he is chief medical officer for Lazarus AI.
The handheld wireless tool, which was developed by Miami-based DermaSensor Inc., operates on battery power, uses spectroscopy and algorithms to evaluate skin lesions for potential cancer in a matter of seconds, and is intended for use by primary care physicians. After the device completes the scan of a lesion, a result of “investigate further” (positive result) suggests further evaluation through a referral to a dermatologist, while “monitor” (negative result) suggests that there is no immediate need for a referral to a dermatologist.
In a pivotal trial of the device that evaluated 224 high risk lesions at 18 primary care study sites in the United States and 4 in Australia, the device had an overall sensitivity of 95.5% for detecting malignancy.
In a more recent validation study funded by DermaSensor, investigators tested 333 lesions at four U.S. dermatology offices and found that the overall device sensitivity was 97.04%, with subgroup sensitivity of 96.67% for melanoma, 97.22% for basal cell carcinoma, and 97.01% for squamous cell carcinoma. Overall specificity of the device was 26.22%.
The study authors, led by Tallahassee, Fla.–based dermatologist Armand B. Cognetta Jr., MD, concluded that DermaSensor’s rapid clinical analysis of lesions “allows for its easy integration into clinical practice infrastructures. Proper use of this device may aid in the reduction of morbidity and mortality associated with skin cancer through expedited and enhanced detection and intervention.”
According to marketing material from the DermaSensor website, the device’s AI algorithm was developed and validated with more than 20,000 scans, composed of more than 4,000 benign and malignant lesions. In a statement about the clearance, the FDA emphasized that the device “should not be used as the sole diagnostic criterion nor to confirm a diagnosis of skin cancer.” The agency is requiring that the manufacturer “conduct additional post-market clinical validation performance testing of the DermaSensor device in patients from demographic groups representative of the U.S. population, including populations who had limited representation of melanomas in the premarket studies, due to their having a relatively low incidence of the disease.”
According to a spokesperson for DermaSensor, pricing for the device is based on a subscription model: $199 per month for five patients or $399 per month for unlimited use. DermaSensor is currently commercially available in Europe and Australia.
Asked to comment, Vishal A. Patel, MD, director of cutaneous oncology at the George Washington Cancer Center, Washington, said that the FDA clearance of DermaSensor highlights the growing appreciation of AI-driven diagnostic support for primary care providers and dermatologists. "Skin cancers are a growing epidemic in the US and the ability to accurately identify potential suspicious lesions without immediately reaching for the scalpel is invaluable," Patel told this news organization. He was not involved with DermSensor studies.
"Furthermore, this tool can help address the shortage of dermatologists and long wait times by helping primary care providers accurately risk-stratify patients and identify those who need to be seen immediately for potential biopsy and expert care," he added. "However, just like with any new technology, we must use caution to not overutilize this tool," which he said, could "lead to overdiagnosis and overtreatment of early or innocuous lesions that are better managed with empiric field treatments."
Dr. Cognetta was a paid investigator for the study.
Dr. Patel disclosed that he is chief medical officer for Lazarus AI.
The handheld wireless tool, which was developed by Miami-based DermaSensor Inc., operates on battery power, uses spectroscopy and algorithms to evaluate skin lesions for potential cancer in a matter of seconds, and is intended for use by primary care physicians. After the device completes the scan of a lesion, a result of “investigate further” (positive result) suggests further evaluation through a referral to a dermatologist, while “monitor” (negative result) suggests that there is no immediate need for a referral to a dermatologist.
In a pivotal trial of the device that evaluated 224 high risk lesions at 18 primary care study sites in the United States and 4 in Australia, the device had an overall sensitivity of 95.5% for detecting malignancy.
In a more recent validation study funded by DermaSensor, investigators tested 333 lesions at four U.S. dermatology offices and found that the overall device sensitivity was 97.04%, with subgroup sensitivity of 96.67% for melanoma, 97.22% for basal cell carcinoma, and 97.01% for squamous cell carcinoma. Overall specificity of the device was 26.22%.
The study authors, led by Tallahassee, Fla.–based dermatologist Armand B. Cognetta Jr., MD, concluded that DermaSensor’s rapid clinical analysis of lesions “allows for its easy integration into clinical practice infrastructures. Proper use of this device may aid in the reduction of morbidity and mortality associated with skin cancer through expedited and enhanced detection and intervention.”
According to marketing material from the DermaSensor website, the device’s AI algorithm was developed and validated with more than 20,000 scans, composed of more than 4,000 benign and malignant lesions. In a statement about the clearance, the FDA emphasized that the device “should not be used as the sole diagnostic criterion nor to confirm a diagnosis of skin cancer.” The agency is requiring that the manufacturer “conduct additional post-market clinical validation performance testing of the DermaSensor device in patients from demographic groups representative of the U.S. population, including populations who had limited representation of melanomas in the premarket studies, due to their having a relatively low incidence of the disease.”
According to a spokesperson for DermaSensor, pricing for the device is based on a subscription model: $199 per month for five patients or $399 per month for unlimited use. DermaSensor is currently commercially available in Europe and Australia.
Asked to comment, Vishal A. Patel, MD, director of cutaneous oncology at the George Washington Cancer Center, Washington, said that the FDA clearance of DermaSensor highlights the growing appreciation of AI-driven diagnostic support for primary care providers and dermatologists. "Skin cancers are a growing epidemic in the US and the ability to accurately identify potential suspicious lesions without immediately reaching for the scalpel is invaluable," Patel told this news organization. He was not involved with DermSensor studies.
"Furthermore, this tool can help address the shortage of dermatologists and long wait times by helping primary care providers accurately risk-stratify patients and identify those who need to be seen immediately for potential biopsy and expert care," he added. "However, just like with any new technology, we must use caution to not overutilize this tool," which he said, could "lead to overdiagnosis and overtreatment of early or innocuous lesions that are better managed with empiric field treatments."
Dr. Cognetta was a paid investigator for the study.
Dr. Patel disclosed that he is chief medical officer for Lazarus AI.
FDA Rejects GI Cancer Drug Over Manufacturing Issues
the company announced January 8.
The monoclonal antibody was under priority review as the first agent specifically for locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma that is claudin 18.2-positive. Overexpression of claudin 18.2 in gastric cancer cells is associated with tumor growth and progression.
The FDA, however, could not approve zolbetuximab by the planned decision date of January 12, 2024, because of “unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility for zolbetuximab,” according to the company press release.
Astellas “is working closely with the FDA and the third-party manufacturer to establish a timeline to quickly resolve” the issues, the company said.
Astellas also clarified that the FDA isn’t asking for additional efficacy and safety data. In phase 3 testing, zolbetuximab improved median progression-free and overall survival by about 2-3 months over chemotherapy alone.
If zolbetuximab is approved, “pathologists will have to be facile with claudin 18.2 testing as a companion diagnostic before [it] can be used,” Mark Lewis, MD, a gastrointestinal oncologist at Intermountain Healthcare in Murray, Utah, told this news organization.
The agent is also under review in Japan, Europe, and China.
A version of this article appeared on Medscape.com.
the company announced January 8.
The monoclonal antibody was under priority review as the first agent specifically for locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma that is claudin 18.2-positive. Overexpression of claudin 18.2 in gastric cancer cells is associated with tumor growth and progression.
The FDA, however, could not approve zolbetuximab by the planned decision date of January 12, 2024, because of “unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility for zolbetuximab,” according to the company press release.
Astellas “is working closely with the FDA and the third-party manufacturer to establish a timeline to quickly resolve” the issues, the company said.
Astellas also clarified that the FDA isn’t asking for additional efficacy and safety data. In phase 3 testing, zolbetuximab improved median progression-free and overall survival by about 2-3 months over chemotherapy alone.
If zolbetuximab is approved, “pathologists will have to be facile with claudin 18.2 testing as a companion diagnostic before [it] can be used,” Mark Lewis, MD, a gastrointestinal oncologist at Intermountain Healthcare in Murray, Utah, told this news organization.
The agent is also under review in Japan, Europe, and China.
A version of this article appeared on Medscape.com.
the company announced January 8.
The monoclonal antibody was under priority review as the first agent specifically for locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma that is claudin 18.2-positive. Overexpression of claudin 18.2 in gastric cancer cells is associated with tumor growth and progression.
The FDA, however, could not approve zolbetuximab by the planned decision date of January 12, 2024, because of “unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility for zolbetuximab,” according to the company press release.
Astellas “is working closely with the FDA and the third-party manufacturer to establish a timeline to quickly resolve” the issues, the company said.
Astellas also clarified that the FDA isn’t asking for additional efficacy and safety data. In phase 3 testing, zolbetuximab improved median progression-free and overall survival by about 2-3 months over chemotherapy alone.
If zolbetuximab is approved, “pathologists will have to be facile with claudin 18.2 testing as a companion diagnostic before [it] can be used,” Mark Lewis, MD, a gastrointestinal oncologist at Intermountain Healthcare in Murray, Utah, told this news organization.
The agent is also under review in Japan, Europe, and China.
A version of this article appeared on Medscape.com.
FDA Gives Nod to Berdazimer Gel for Molluscum Contagiosum
On January 5, the Food and Drug Administration (FDA) approved berdazimer gel 10.3% for the treatment of molluscum contagiosum (MC) in adults and children aged 1 year or older.
Approval of berdazimer, a topical nitric oxide–releasing agent, was based largely on a 12-week pivotal phase 3 trial known as B-SIMPLE4, in which 891 patients with a mean age of 6.6 years (range, 0.9-47.5 years) were randomly assigned to treatment with berdazimer gel 10.3% or a vehicle gel applied in a thin layer to all lesions once daily. At 12 weeks, 32.4% of patients in the berdazimer group achieved complete clearance of MC lesions compared with 19.7% of those in the vehicle group (P < .001).
Only 4.1% of patients on berdazimer and 0.7% of those on the vehicle experienced adverse events that led to discontinuation of treatment. The most common adverse events in both groups were application-site pain and erythema, and most of these were mild or moderate.
According to a press release announcing the approval from Ligand Pharmaceuticals, which acquired berdazimer topical gel from Novan in September 2023, the development makes berdazimer topical gel 10.3% the first and only topical prescription medication that can be applied by patients, parents, or caregivers at home; outside of a physician›s office; or outside of other medical settings to treat MC. Nitric oxide has been shown to have antiviral effects, although the mechanism of action of berdazimer for treating molluscum “is unknown,” the company said in the release.
The drug will be marketed under the name Zelsuvmi and is expected to be available in the second half of 2024.
On July 21, 2023, topical cantharidin became the first approved treatment of MC for adults and pediatric patients aged 2 years or older, with the FDA approval of a drug-device combination (Ycanth) that contains a formulation of cantharidin solution 0.7% and is administered by healthcare professionals.
A version of this article appeared on Medscape.com.
On January 5, the Food and Drug Administration (FDA) approved berdazimer gel 10.3% for the treatment of molluscum contagiosum (MC) in adults and children aged 1 year or older.
Approval of berdazimer, a topical nitric oxide–releasing agent, was based largely on a 12-week pivotal phase 3 trial known as B-SIMPLE4, in which 891 patients with a mean age of 6.6 years (range, 0.9-47.5 years) were randomly assigned to treatment with berdazimer gel 10.3% or a vehicle gel applied in a thin layer to all lesions once daily. At 12 weeks, 32.4% of patients in the berdazimer group achieved complete clearance of MC lesions compared with 19.7% of those in the vehicle group (P < .001).
Only 4.1% of patients on berdazimer and 0.7% of those on the vehicle experienced adverse events that led to discontinuation of treatment. The most common adverse events in both groups were application-site pain and erythema, and most of these were mild or moderate.
According to a press release announcing the approval from Ligand Pharmaceuticals, which acquired berdazimer topical gel from Novan in September 2023, the development makes berdazimer topical gel 10.3% the first and only topical prescription medication that can be applied by patients, parents, or caregivers at home; outside of a physician›s office; or outside of other medical settings to treat MC. Nitric oxide has been shown to have antiviral effects, although the mechanism of action of berdazimer for treating molluscum “is unknown,” the company said in the release.
The drug will be marketed under the name Zelsuvmi and is expected to be available in the second half of 2024.
On July 21, 2023, topical cantharidin became the first approved treatment of MC for adults and pediatric patients aged 2 years or older, with the FDA approval of a drug-device combination (Ycanth) that contains a formulation of cantharidin solution 0.7% and is administered by healthcare professionals.
A version of this article appeared on Medscape.com.
On January 5, the Food and Drug Administration (FDA) approved berdazimer gel 10.3% for the treatment of molluscum contagiosum (MC) in adults and children aged 1 year or older.
Approval of berdazimer, a topical nitric oxide–releasing agent, was based largely on a 12-week pivotal phase 3 trial known as B-SIMPLE4, in which 891 patients with a mean age of 6.6 years (range, 0.9-47.5 years) were randomly assigned to treatment with berdazimer gel 10.3% or a vehicle gel applied in a thin layer to all lesions once daily. At 12 weeks, 32.4% of patients in the berdazimer group achieved complete clearance of MC lesions compared with 19.7% of those in the vehicle group (P < .001).
Only 4.1% of patients on berdazimer and 0.7% of those on the vehicle experienced adverse events that led to discontinuation of treatment. The most common adverse events in both groups were application-site pain and erythema, and most of these were mild or moderate.
According to a press release announcing the approval from Ligand Pharmaceuticals, which acquired berdazimer topical gel from Novan in September 2023, the development makes berdazimer topical gel 10.3% the first and only topical prescription medication that can be applied by patients, parents, or caregivers at home; outside of a physician›s office; or outside of other medical settings to treat MC. Nitric oxide has been shown to have antiviral effects, although the mechanism of action of berdazimer for treating molluscum “is unknown,” the company said in the release.
The drug will be marketed under the name Zelsuvmi and is expected to be available in the second half of 2024.
On July 21, 2023, topical cantharidin became the first approved treatment of MC for adults and pediatric patients aged 2 years or older, with the FDA approval of a drug-device combination (Ycanth) that contains a formulation of cantharidin solution 0.7% and is administered by healthcare professionals.
A version of this article appeared on Medscape.com.
FDA Investigates Three Side Effects Reported With Weight Loss Drugs
or two other health problems.
A new FDA report listed potential links between the medications and alopecia, aspiration, or suicidal ideation, CBS News reported. The investigation centers on reports of the health problems among people taking GLP-1 receptor agonists, some of which are Ozempic, Wegovy, Mounjaro, and Zepbound. The drugs are used to treat diabetes and overweight or obesity.
An investigation by the FDA doesn’t mean that the FDA has concluded a risk exists, the FDA’s webpage for risk evaluation cautions.
“It means that FDA has identified a potential safety issue, but it does not mean that FDA has identified a causal relationship between the drug and the listed risk,” the FDA site states.
Possible next steps after an investigation could include updating drug labels with new information, putting a risk management plan in place to prevent or manage the health risks, or gathering more information.
“The FDA monitors the safety of drugs throughout their life cycle,” even after the drugs are approved. In addition, the FDA uses “surveillance and risk assessment programs to identify and evaluate adverse events that did not appear during the drug development process,” FDA spokesperson Chanapa Tantibanchachai said in an email published by multiple news outlets.
Although an investigation may lead to no changes in how a drug is regulated by the FDA, this isn’t the first time that the popular medicines have landed on the FDA’s radar for safety reevaluation. Last year, the label for the drug Ozempic was updated to acknowledge reports of intestinal obstructions, CBS News reported.
European regulators are also looking into reports of suicidal thoughts among people taking GLP-1 receptor agonists, although no link has been established.
Concerns about aspiration during surgery resulted in the American Society of Anesthesiologists advising in June that people should stop taking GLP-1 receptor agonists before they have elective surgeries.
“While there is currently a lack of scientific data on how GLP-1 receptor agonists affect patients having surgery and interact with anesthesia, we’ve received anecdotal reports that the delay in stomach emptying could be associated with an increased risk of regurgitation and aspiration of food into the airways and lungs during general anesthesia and deep sedation,” the society’s president, Michael W. Champeau, MD, said in a statement at the time.
According to CBS News, the FDA’s drug reporting system links the medications to 201 reports of suicide or suicidal ideation, 18 reports that mention aspiration, and 422 reports that mention alopecia.
Novo Nordisk, whose portfolio includes Wegovy and Ozempic, told CNN that it works with the FDA to monitor safety and is aware of the reports of side effects.
“Novo Nordisk stands behind the safety and efficacy of all of our GLP-1RA medicines when they are used as indicated and when they are taken under the care of a licensed healthcare professional,” the company said in a statement to CNN.
A spokesperson for Eli Lilly, which makes Mounjaro and Zepbound, told CBS News in a statement, “Currently, the FDA is reviewing data on certain potential risks for GLP-1 receptor agonist medicines. Patient safety is our priority, and we are collaborating with the FDA on these potential signals.”
A version of this article appeared on WebMD.com .
or two other health problems.
A new FDA report listed potential links between the medications and alopecia, aspiration, or suicidal ideation, CBS News reported. The investigation centers on reports of the health problems among people taking GLP-1 receptor agonists, some of which are Ozempic, Wegovy, Mounjaro, and Zepbound. The drugs are used to treat diabetes and overweight or obesity.
An investigation by the FDA doesn’t mean that the FDA has concluded a risk exists, the FDA’s webpage for risk evaluation cautions.
“It means that FDA has identified a potential safety issue, but it does not mean that FDA has identified a causal relationship between the drug and the listed risk,” the FDA site states.
Possible next steps after an investigation could include updating drug labels with new information, putting a risk management plan in place to prevent or manage the health risks, or gathering more information.
“The FDA monitors the safety of drugs throughout their life cycle,” even after the drugs are approved. In addition, the FDA uses “surveillance and risk assessment programs to identify and evaluate adverse events that did not appear during the drug development process,” FDA spokesperson Chanapa Tantibanchachai said in an email published by multiple news outlets.
Although an investigation may lead to no changes in how a drug is regulated by the FDA, this isn’t the first time that the popular medicines have landed on the FDA’s radar for safety reevaluation. Last year, the label for the drug Ozempic was updated to acknowledge reports of intestinal obstructions, CBS News reported.
European regulators are also looking into reports of suicidal thoughts among people taking GLP-1 receptor agonists, although no link has been established.
Concerns about aspiration during surgery resulted in the American Society of Anesthesiologists advising in June that people should stop taking GLP-1 receptor agonists before they have elective surgeries.
“While there is currently a lack of scientific data on how GLP-1 receptor agonists affect patients having surgery and interact with anesthesia, we’ve received anecdotal reports that the delay in stomach emptying could be associated with an increased risk of regurgitation and aspiration of food into the airways and lungs during general anesthesia and deep sedation,” the society’s president, Michael W. Champeau, MD, said in a statement at the time.
According to CBS News, the FDA’s drug reporting system links the medications to 201 reports of suicide or suicidal ideation, 18 reports that mention aspiration, and 422 reports that mention alopecia.
Novo Nordisk, whose portfolio includes Wegovy and Ozempic, told CNN that it works with the FDA to monitor safety and is aware of the reports of side effects.
“Novo Nordisk stands behind the safety and efficacy of all of our GLP-1RA medicines when they are used as indicated and when they are taken under the care of a licensed healthcare professional,” the company said in a statement to CNN.
A spokesperson for Eli Lilly, which makes Mounjaro and Zepbound, told CBS News in a statement, “Currently, the FDA is reviewing data on certain potential risks for GLP-1 receptor agonist medicines. Patient safety is our priority, and we are collaborating with the FDA on these potential signals.”
A version of this article appeared on WebMD.com .
or two other health problems.
A new FDA report listed potential links between the medications and alopecia, aspiration, or suicidal ideation, CBS News reported. The investigation centers on reports of the health problems among people taking GLP-1 receptor agonists, some of which are Ozempic, Wegovy, Mounjaro, and Zepbound. The drugs are used to treat diabetes and overweight or obesity.
An investigation by the FDA doesn’t mean that the FDA has concluded a risk exists, the FDA’s webpage for risk evaluation cautions.
“It means that FDA has identified a potential safety issue, but it does not mean that FDA has identified a causal relationship between the drug and the listed risk,” the FDA site states.
Possible next steps after an investigation could include updating drug labels with new information, putting a risk management plan in place to prevent or manage the health risks, or gathering more information.
“The FDA monitors the safety of drugs throughout their life cycle,” even after the drugs are approved. In addition, the FDA uses “surveillance and risk assessment programs to identify and evaluate adverse events that did not appear during the drug development process,” FDA spokesperson Chanapa Tantibanchachai said in an email published by multiple news outlets.
Although an investigation may lead to no changes in how a drug is regulated by the FDA, this isn’t the first time that the popular medicines have landed on the FDA’s radar for safety reevaluation. Last year, the label for the drug Ozempic was updated to acknowledge reports of intestinal obstructions, CBS News reported.
European regulators are also looking into reports of suicidal thoughts among people taking GLP-1 receptor agonists, although no link has been established.
Concerns about aspiration during surgery resulted in the American Society of Anesthesiologists advising in June that people should stop taking GLP-1 receptor agonists before they have elective surgeries.
“While there is currently a lack of scientific data on how GLP-1 receptor agonists affect patients having surgery and interact with anesthesia, we’ve received anecdotal reports that the delay in stomach emptying could be associated with an increased risk of regurgitation and aspiration of food into the airways and lungs during general anesthesia and deep sedation,” the society’s president, Michael W. Champeau, MD, said in a statement at the time.
According to CBS News, the FDA’s drug reporting system links the medications to 201 reports of suicide or suicidal ideation, 18 reports that mention aspiration, and 422 reports that mention alopecia.
Novo Nordisk, whose portfolio includes Wegovy and Ozempic, told CNN that it works with the FDA to monitor safety and is aware of the reports of side effects.
“Novo Nordisk stands behind the safety and efficacy of all of our GLP-1RA medicines when they are used as indicated and when they are taken under the care of a licensed healthcare professional,” the company said in a statement to CNN.
A spokesperson for Eli Lilly, which makes Mounjaro and Zepbound, told CBS News in a statement, “Currently, the FDA is reviewing data on certain potential risks for GLP-1 receptor agonist medicines. Patient safety is our priority, and we are collaborating with the FDA on these potential signals.”
A version of this article appeared on WebMD.com .