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CHMP supports approval of denosumab in MM
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the current indication for denosumab (XGEVA®).
The CHMP said the drug should be approved for use in preventing skeletal related events (SREs) in adults with advanced malignancies involving bone, which includes multiple myeloma (MM).
Denosumab is currently approved in Europe to prevent SREs—defined as radiation to bone, pathologic fracture, surgery to bone, and spinal cord compression—in adults with bone metastases from solid tumors.
The drug is also approved for the treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity.
The CHMP’s opinion on expanding the indication for denosumab will be reviewed by the European Commission (EC).
If the EC agrees with the CHMP, the commission will grant a centralized marketing authorization that will be valid in the European Union. Norway, Iceland, and Liechtenstein will make corresponding decisions on the basis of the EC’s decision.
The EC typically makes a decision within 67 days of the CHMP’s recommendation.
The CHMP’s recommendation for denosumab is based on data from the ’482 study, which were recently published in The Lancet Oncology.
In this phase 3 trial, denosumab proved non-inferior to zoledronic acid for delaying SREs in patients with newly diagnosed MM and bone disease.
Researchers randomized 1718 patients to receive subcutaneous denosumab at 120 mg and intravenous placebo every 4 weeks (n=859) or intravenous zoledronic acid at 4 mg (adjusted for renal function at baseline) and subcutaneous placebo every 4 weeks (n=859). All patients also received investigators’ choice of first-line MM therapy.
The median time to first on-study SRE was 22.8 months for patients in the denosumab arm and 24 months for those in the zoledronic acid arm (hazard ratio=0.98; 95% confidence interval: 0.85-1.14; P non-inferiority=0.010).
There were fewer renal treatment-emergent adverse events in the denosumab arm than the zoledronic acid arm—10% and 17%, respectively.
But there were more hypocalcemia adverse events in the denosumab arm than the zoledronic acid arm—17% and 12%, respectively.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the current indication for denosumab (XGEVA®).
The CHMP said the drug should be approved for use in preventing skeletal related events (SREs) in adults with advanced malignancies involving bone, which includes multiple myeloma (MM).
Denosumab is currently approved in Europe to prevent SREs—defined as radiation to bone, pathologic fracture, surgery to bone, and spinal cord compression—in adults with bone metastases from solid tumors.
The drug is also approved for the treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity.
The CHMP’s opinion on expanding the indication for denosumab will be reviewed by the European Commission (EC).
If the EC agrees with the CHMP, the commission will grant a centralized marketing authorization that will be valid in the European Union. Norway, Iceland, and Liechtenstein will make corresponding decisions on the basis of the EC’s decision.
The EC typically makes a decision within 67 days of the CHMP’s recommendation.
The CHMP’s recommendation for denosumab is based on data from the ’482 study, which were recently published in The Lancet Oncology.
In this phase 3 trial, denosumab proved non-inferior to zoledronic acid for delaying SREs in patients with newly diagnosed MM and bone disease.
Researchers randomized 1718 patients to receive subcutaneous denosumab at 120 mg and intravenous placebo every 4 weeks (n=859) or intravenous zoledronic acid at 4 mg (adjusted for renal function at baseline) and subcutaneous placebo every 4 weeks (n=859). All patients also received investigators’ choice of first-line MM therapy.
The median time to first on-study SRE was 22.8 months for patients in the denosumab arm and 24 months for those in the zoledronic acid arm (hazard ratio=0.98; 95% confidence interval: 0.85-1.14; P non-inferiority=0.010).
There were fewer renal treatment-emergent adverse events in the denosumab arm than the zoledronic acid arm—10% and 17%, respectively.
But there were more hypocalcemia adverse events in the denosumab arm than the zoledronic acid arm—17% and 12%, respectively.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the current indication for denosumab (XGEVA®).
The CHMP said the drug should be approved for use in preventing skeletal related events (SREs) in adults with advanced malignancies involving bone, which includes multiple myeloma (MM).
Denosumab is currently approved in Europe to prevent SREs—defined as radiation to bone, pathologic fracture, surgery to bone, and spinal cord compression—in adults with bone metastases from solid tumors.
The drug is also approved for the treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity.
The CHMP’s opinion on expanding the indication for denosumab will be reviewed by the European Commission (EC).
If the EC agrees with the CHMP, the commission will grant a centralized marketing authorization that will be valid in the European Union. Norway, Iceland, and Liechtenstein will make corresponding decisions on the basis of the EC’s decision.
The EC typically makes a decision within 67 days of the CHMP’s recommendation.
The CHMP’s recommendation for denosumab is based on data from the ’482 study, which were recently published in The Lancet Oncology.
In this phase 3 trial, denosumab proved non-inferior to zoledronic acid for delaying SREs in patients with newly diagnosed MM and bone disease.
Researchers randomized 1718 patients to receive subcutaneous denosumab at 120 mg and intravenous placebo every 4 weeks (n=859) or intravenous zoledronic acid at 4 mg (adjusted for renal function at baseline) and subcutaneous placebo every 4 weeks (n=859). All patients also received investigators’ choice of first-line MM therapy.
The median time to first on-study SRE was 22.8 months for patients in the denosumab arm and 24 months for those in the zoledronic acid arm (hazard ratio=0.98; 95% confidence interval: 0.85-1.14; P non-inferiority=0.010).
There were fewer renal treatment-emergent adverse events in the denosumab arm than the zoledronic acid arm—10% and 17%, respectively.
But there were more hypocalcemia adverse events in the denosumab arm than the zoledronic acid arm—17% and 12%, respectively.
Drug receives orphan designation for MM
The US Food and Drug Administration (FDA) has granted orphan designation to PT-112 as a treatment for multiple myeloma (MM).
PT-112 is a small-molecule conjugate of pyrophosphate and platinum that promotes apoptosis with damage-associated molecular patterns, leading to downstream T-cell recruitment in the tumor microenvironment.
PT-112 is currently under investigation in a phase 1/2 study of patients with relapsed or refractory MM (NCT03288480).
Phosplatin Therapeutics LLC, the company developing PT-112, has enrolled the first cohort of patients in this trial.
In preclinical experiments, PT-112 demonstrated synergy with lenalidomide and bortezomib in RPMI-8226 cells and dexamethasone-resistant MM1R cells.
Single-agent PT-112 produced responses in mice with established MM. Researchers said PT-112 had “pronounced” activity against bortezomib-refractory Vk12598 tumors, which significantly improved overall survival in the mice.
This research was presented at the 2017 ASH Annual Meeting (abstract 1797).
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
The US Food and Drug Administration (FDA) has granted orphan designation to PT-112 as a treatment for multiple myeloma (MM).
PT-112 is a small-molecule conjugate of pyrophosphate and platinum that promotes apoptosis with damage-associated molecular patterns, leading to downstream T-cell recruitment in the tumor microenvironment.
PT-112 is currently under investigation in a phase 1/2 study of patients with relapsed or refractory MM (NCT03288480).
Phosplatin Therapeutics LLC, the company developing PT-112, has enrolled the first cohort of patients in this trial.
In preclinical experiments, PT-112 demonstrated synergy with lenalidomide and bortezomib in RPMI-8226 cells and dexamethasone-resistant MM1R cells.
Single-agent PT-112 produced responses in mice with established MM. Researchers said PT-112 had “pronounced” activity against bortezomib-refractory Vk12598 tumors, which significantly improved overall survival in the mice.
This research was presented at the 2017 ASH Annual Meeting (abstract 1797).
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
The US Food and Drug Administration (FDA) has granted orphan designation to PT-112 as a treatment for multiple myeloma (MM).
PT-112 is a small-molecule conjugate of pyrophosphate and platinum that promotes apoptosis with damage-associated molecular patterns, leading to downstream T-cell recruitment in the tumor microenvironment.
PT-112 is currently under investigation in a phase 1/2 study of patients with relapsed or refractory MM (NCT03288480).
Phosplatin Therapeutics LLC, the company developing PT-112, has enrolled the first cohort of patients in this trial.
In preclinical experiments, PT-112 demonstrated synergy with lenalidomide and bortezomib in RPMI-8226 cells and dexamethasone-resistant MM1R cells.
Single-agent PT-112 produced responses in mice with established MM. Researchers said PT-112 had “pronounced” activity against bortezomib-refractory Vk12598 tumors, which significantly improved overall survival in the mice.
This research was presented at the 2017 ASH Annual Meeting (abstract 1797).
About orphan designation
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
Factor IX product launched in US
The recombinant, GlycoPEGylated coagulation factor IX product Rebinyn® is now available in the US for the treatment of patients with hemophilia B.
Last May, Rebinyn was approved by the US Food and Drug Administration for on-demand treatment and control of bleeding episodes as well as perioperative management of bleeding in adults and children with hemophilia B.
The product is not approved for routine prophylaxis or immune tolerance induction in hemophilia B patients.
The full prescribing information is available at www.Rebinyn.com.
Rebinyn is also approved for use in the European Union, where it is known as nonacog beta pegol or by the brand name Refixia.
There, the product is approved for use as prophylaxis, for on-demand treatment of bleeding, and for control of bleeding related to surgical procedures in adolescents (older than 12 years of age) and adults with hemophilia B.
Trial results
The US and European approvals of nonacog beta pegol (N9-GP) were based on results from the paradigm™ clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Paradigm 4 was an extension trial enrolling patients who had participated in the phase 3 trials paradigm 2 and paradigm 3.
In paradigm 2, researchers assessed N9-GP as treatment and prophylaxis in previously treated patients with hemophilia B. In paradigm 3, researchers assessed N9-GP in hemophilia B patients undergoing surgical procedures.
Paradigm 4 included 71 patients (ages 13 to 70) who continued to receive N9-GP as on-demand treatment (40 IU/kg for mild/moderate bleeds and 80 IU/kg for severe bleeds) or prophylaxis (10 IU/kg or 40 IU/kg once-weekly). Sixty-five patients completed treatment.
Safety
None of the patients developed factor IX inhibitors. Two patients had transient binding antibodies to N9-GP, but there was no sign that these antibodies had an inhibitory effect.
Four patients developed anti-CHO antibodies, but only 2 of these patients were still positive for these antibodies at the end of the trial.
There were a total of 155 adverse events. However, only 4 of these (in 3 patients) were considered possibly or probably related to N9-GP.
These events consisted of an injection site rash in 1 patient, 2 overdoses in 1 patient, and neutropenia in 1 patient. The rash and neutropenia resolved, and the patient who overdosed recovered without complications.
Efficacy
The researchers said the success rate for the treatment of reported bleeds was 94.6%. Most bleeds (87.9%) were resolved with a single injection of N9-GP, but 9.2% required 2 injections, and 2.9% required 3 or 4 injections.
The median annualized bleeding rate for patients on prophylaxis was 1.05 (interquartile range [IQR], 0.00–2.20) overall. It was 1.36 (IQR, 0.00-2.23) for the 10 IU/kg arm and 1.00 (IQR, 0.00-2.03) for the 40 IU/kg arm.
There were 14 patients on prophylaxis who underwent 23 minor surgical procedures.
The hemostatic response was considered “excellent” (better than expected/predicted for the procedure in question) in 19 procedures and “good” (as expected) in 2 procedures. In the remaining 2 procedures, hemostatic responses were not determined. 
The recombinant, GlycoPEGylated coagulation factor IX product Rebinyn® is now available in the US for the treatment of patients with hemophilia B.
Last May, Rebinyn was approved by the US Food and Drug Administration for on-demand treatment and control of bleeding episodes as well as perioperative management of bleeding in adults and children with hemophilia B.
The product is not approved for routine prophylaxis or immune tolerance induction in hemophilia B patients.
The full prescribing information is available at www.Rebinyn.com.
Rebinyn is also approved for use in the European Union, where it is known as nonacog beta pegol or by the brand name Refixia.
There, the product is approved for use as prophylaxis, for on-demand treatment of bleeding, and for control of bleeding related to surgical procedures in adolescents (older than 12 years of age) and adults with hemophilia B.
Trial results
The US and European approvals of nonacog beta pegol (N9-GP) were based on results from the paradigm™ clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Paradigm 4 was an extension trial enrolling patients who had participated in the phase 3 trials paradigm 2 and paradigm 3.
In paradigm 2, researchers assessed N9-GP as treatment and prophylaxis in previously treated patients with hemophilia B. In paradigm 3, researchers assessed N9-GP in hemophilia B patients undergoing surgical procedures.
Paradigm 4 included 71 patients (ages 13 to 70) who continued to receive N9-GP as on-demand treatment (40 IU/kg for mild/moderate bleeds and 80 IU/kg for severe bleeds) or prophylaxis (10 IU/kg or 40 IU/kg once-weekly). Sixty-five patients completed treatment.
Safety
None of the patients developed factor IX inhibitors. Two patients had transient binding antibodies to N9-GP, but there was no sign that these antibodies had an inhibitory effect.
Four patients developed anti-CHO antibodies, but only 2 of these patients were still positive for these antibodies at the end of the trial.
There were a total of 155 adverse events. However, only 4 of these (in 3 patients) were considered possibly or probably related to N9-GP.
These events consisted of an injection site rash in 1 patient, 2 overdoses in 1 patient, and neutropenia in 1 patient. The rash and neutropenia resolved, and the patient who overdosed recovered without complications.
Efficacy
The researchers said the success rate for the treatment of reported bleeds was 94.6%. Most bleeds (87.9%) were resolved with a single injection of N9-GP, but 9.2% required 2 injections, and 2.9% required 3 or 4 injections.
The median annualized bleeding rate for patients on prophylaxis was 1.05 (interquartile range [IQR], 0.00–2.20) overall. It was 1.36 (IQR, 0.00-2.23) for the 10 IU/kg arm and 1.00 (IQR, 0.00-2.03) for the 40 IU/kg arm.
There were 14 patients on prophylaxis who underwent 23 minor surgical procedures.
The hemostatic response was considered “excellent” (better than expected/predicted for the procedure in question) in 19 procedures and “good” (as expected) in 2 procedures. In the remaining 2 procedures, hemostatic responses were not determined.
The recombinant, GlycoPEGylated coagulation factor IX product Rebinyn® is now available in the US for the treatment of patients with hemophilia B.
Last May, Rebinyn was approved by the US Food and Drug Administration for on-demand treatment and control of bleeding episodes as well as perioperative management of bleeding in adults and children with hemophilia B.
The product is not approved for routine prophylaxis or immune tolerance induction in hemophilia B patients.
The full prescribing information is available at www.Rebinyn.com.
Rebinyn is also approved for use in the European Union, where it is known as nonacog beta pegol or by the brand name Refixia.
There, the product is approved for use as prophylaxis, for on-demand treatment of bleeding, and for control of bleeding related to surgical procedures in adolescents (older than 12 years of age) and adults with hemophilia B.
Trial results
The US and European approvals of nonacog beta pegol (N9-GP) were based on results from the paradigm™ clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Paradigm 4 was an extension trial enrolling patients who had participated in the phase 3 trials paradigm 2 and paradigm 3.
In paradigm 2, researchers assessed N9-GP as treatment and prophylaxis in previously treated patients with hemophilia B. In paradigm 3, researchers assessed N9-GP in hemophilia B patients undergoing surgical procedures.
Paradigm 4 included 71 patients (ages 13 to 70) who continued to receive N9-GP as on-demand treatment (40 IU/kg for mild/moderate bleeds and 80 IU/kg for severe bleeds) or prophylaxis (10 IU/kg or 40 IU/kg once-weekly). Sixty-five patients completed treatment.
Safety
None of the patients developed factor IX inhibitors. Two patients had transient binding antibodies to N9-GP, but there was no sign that these antibodies had an inhibitory effect.
Four patients developed anti-CHO antibodies, but only 2 of these patients were still positive for these antibodies at the end of the trial.
There were a total of 155 adverse events. However, only 4 of these (in 3 patients) were considered possibly or probably related to N9-GP.
These events consisted of an injection site rash in 1 patient, 2 overdoses in 1 patient, and neutropenia in 1 patient. The rash and neutropenia resolved, and the patient who overdosed recovered without complications.
Efficacy
The researchers said the success rate for the treatment of reported bleeds was 94.6%. Most bleeds (87.9%) were resolved with a single injection of N9-GP, but 9.2% required 2 injections, and 2.9% required 3 or 4 injections.
The median annualized bleeding rate for patients on prophylaxis was 1.05 (interquartile range [IQR], 0.00–2.20) overall. It was 1.36 (IQR, 0.00-2.23) for the 10 IU/kg arm and 1.00 (IQR, 0.00-2.03) for the 40 IU/kg arm.
There were 14 patients on prophylaxis who underwent 23 minor surgical procedures.
The hemostatic response was considered “excellent” (better than expected/predicted for the procedure in question) in 19 procedures and “good” (as expected) in 2 procedures. In the remaining 2 procedures, hemostatic responses were not determined.
Azacitidine now available in China
Azacitidine for injection (Vidaza®) is now available in China.
The nucleoside metabolic inhibitor was approved in China to treat patients with intermediate-2/high-risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) with 20% to 30% bone marrow blasts, and chronic myelomonocytic leukemia (CMML).
Azacitidine for injection is marketed in China by BeiGene Ltd. under an exclusive license from Celgene Corporation.
“Vidaza is the only approved hypomethylating agent shown to prolong survival for patients with MDS and the first new treatment for MDS patients approved in China since 2009,” said John V. Oyler, founder, chief executive officer, and chairman of BeiGene.
“We are excited to announce that the first prescription was made in January 2018. From now on, Chinese patients can benefit from Vidaza in hospitals around China.”
Azacitidine was evaluated in a global phase 3 trial of patients with intermediate-2- and high-risk MDS, CMML, or AML (AZA-001). Results from this trial were published in The Lancet Oncology in 2009.
Patients were randomized to receive azacitidine plus best supportive care (BSC, n=179) or conventional care regimens plus BSC (105 to BSC alone, 49 to low-dose cytarabine, and 25 to chemotherapy with cytarabine and anthracycline).
Azacitidine was given subcutaneously at a dose of 75 mg/m2 daily for 7 consecutive days every 28 days until disease progression, relapse after response, or unacceptable toxicity.
The median overall survival was 24.5 months with azacitidine, compared to 15 months for patients treated with conventional care regimens.
There was a higher hematologic response rate in the azacitidine arm than the conventional care arm—29% and 12%, respectively.
In the azacitidine group, 45% of patients who were dependent on red blood cell transfusions at baseline became transfusion independent, compared with 11% in the conventional care group.
Forty-six percent of patients in the azacitidine arm and 63% in the conventional care arm died.
Grade 3/4 hematologic toxicity (in the azacitidine and conventional care arms, respectively) included neutropenia (91% and 76%), thrombocytopenia (85% and 80%), and anemia (57% and 68%).
Azacitidine for injection (Vidaza®) is now available in China.
The nucleoside metabolic inhibitor was approved in China to treat patients with intermediate-2/high-risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) with 20% to 30% bone marrow blasts, and chronic myelomonocytic leukemia (CMML).
Azacitidine for injection is marketed in China by BeiGene Ltd. under an exclusive license from Celgene Corporation.
“Vidaza is the only approved hypomethylating agent shown to prolong survival for patients with MDS and the first new treatment for MDS patients approved in China since 2009,” said John V. Oyler, founder, chief executive officer, and chairman of BeiGene.
“We are excited to announce that the first prescription was made in January 2018. From now on, Chinese patients can benefit from Vidaza in hospitals around China.”
Azacitidine was evaluated in a global phase 3 trial of patients with intermediate-2- and high-risk MDS, CMML, or AML (AZA-001). Results from this trial were published in The Lancet Oncology in 2009.
Patients were randomized to receive azacitidine plus best supportive care (BSC, n=179) or conventional care regimens plus BSC (105 to BSC alone, 49 to low-dose cytarabine, and 25 to chemotherapy with cytarabine and anthracycline).
Azacitidine was given subcutaneously at a dose of 75 mg/m2 daily for 7 consecutive days every 28 days until disease progression, relapse after response, or unacceptable toxicity.
The median overall survival was 24.5 months with azacitidine, compared to 15 months for patients treated with conventional care regimens.
There was a higher hematologic response rate in the azacitidine arm than the conventional care arm—29% and 12%, respectively.
In the azacitidine group, 45% of patients who were dependent on red blood cell transfusions at baseline became transfusion independent, compared with 11% in the conventional care group.
Forty-six percent of patients in the azacitidine arm and 63% in the conventional care arm died.
Grade 3/4 hematologic toxicity (in the azacitidine and conventional care arms, respectively) included neutropenia (91% and 76%), thrombocytopenia (85% and 80%), and anemia (57% and 68%).
Azacitidine for injection (Vidaza®) is now available in China.
The nucleoside metabolic inhibitor was approved in China to treat patients with intermediate-2/high-risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) with 20% to 30% bone marrow blasts, and chronic myelomonocytic leukemia (CMML).
Azacitidine for injection is marketed in China by BeiGene Ltd. under an exclusive license from Celgene Corporation.
“Vidaza is the only approved hypomethylating agent shown to prolong survival for patients with MDS and the first new treatment for MDS patients approved in China since 2009,” said John V. Oyler, founder, chief executive officer, and chairman of BeiGene.
“We are excited to announce that the first prescription was made in January 2018. From now on, Chinese patients can benefit from Vidaza in hospitals around China.”
Azacitidine was evaluated in a global phase 3 trial of patients with intermediate-2- and high-risk MDS, CMML, or AML (AZA-001). Results from this trial were published in The Lancet Oncology in 2009.
Patients were randomized to receive azacitidine plus best supportive care (BSC, n=179) or conventional care regimens plus BSC (105 to BSC alone, 49 to low-dose cytarabine, and 25 to chemotherapy with cytarabine and anthracycline).
Azacitidine was given subcutaneously at a dose of 75 mg/m2 daily for 7 consecutive days every 28 days until disease progression, relapse after response, or unacceptable toxicity.
The median overall survival was 24.5 months with azacitidine, compared to 15 months for patients treated with conventional care regimens.
There was a higher hematologic response rate in the azacitidine arm than the conventional care arm—29% and 12%, respectively.
In the azacitidine group, 45% of patients who were dependent on red blood cell transfusions at baseline became transfusion independent, compared with 11% in the conventional care group.
Forty-six percent of patients in the azacitidine arm and 63% in the conventional care arm died.
Grade 3/4 hematologic toxicity (in the azacitidine and conventional care arms, respectively) included neutropenia (91% and 76%), thrombocytopenia (85% and 80%), and anemia (57% and 68%).
FDA expands approved use of ferumoxytol injection
The US Food and Drug Administration (FDA) has expanded the approved indication for ferumoxytol injection (Feraheme®).
The drug is now approved to treat adults with iron deficiency anemia (IDA) who cannot tolerate or have had an unsatisfactory response to oral iron.
Ferumoxytol injection was previously approved by the FDA to treat IDA in adults with chronic kidney disease.
“Iron deficiency anemia is a serious and under-treated health condition which negatively impacts quality of life for millions of people, many of whom do not benefit from or cannot tolerate oral iron therapy,” said Michael Auerbach, MD, of Georgetown University School of Medicine in Washington, DC.
“Physicians now have a new option for patients who meet the broader ferumoxytol injection indication that can be administered in 15 minutes, providing a gram of iron in 2 doses as few as 3 days apart.”
The expanded approval for ferumoxytol injection was supported by a trio of phase 3 trials. All 3 trials included IDA patients who could not tolerate or had an unsatisfactory response to oral iron.
In the first trial (NCT01114139), researchers compared ferumoxytol injection to placebo.
The second trial (NCT01114204) was a comparison of ferumoxytol injection and iron sucrose.
In the third trial (NCT02694978), researchers compared ferumoxytol injection to ferric carboxymaltose injection (Injectafer®).
Details on these trials are included in the prescribing information for ferumoxytol injection, which is available at www.feraheme.com.
The prescribing information includes a boxed warning detailing the risk of fatal and serious hypersensitivity reactions, including anaphylaxis, in patients receiving ferumoxytol injection.
Ferumoxytol injection is a product of AMAG Pharmaceuticals, Inc.
The company has a patient access support program called AMAG Assist™. Uninsured or underinsured patients who need help paying for their ferumoxytol injection prescription can call 844-635-2624 to see if they qualify for help.
The US Food and Drug Administration (FDA) has expanded the approved indication for ferumoxytol injection (Feraheme®).
The drug is now approved to treat adults with iron deficiency anemia (IDA) who cannot tolerate or have had an unsatisfactory response to oral iron.
Ferumoxytol injection was previously approved by the FDA to treat IDA in adults with chronic kidney disease.
“Iron deficiency anemia is a serious and under-treated health condition which negatively impacts quality of life for millions of people, many of whom do not benefit from or cannot tolerate oral iron therapy,” said Michael Auerbach, MD, of Georgetown University School of Medicine in Washington, DC.
“Physicians now have a new option for patients who meet the broader ferumoxytol injection indication that can be administered in 15 minutes, providing a gram of iron in 2 doses as few as 3 days apart.”
The expanded approval for ferumoxytol injection was supported by a trio of phase 3 trials. All 3 trials included IDA patients who could not tolerate or had an unsatisfactory response to oral iron.
In the first trial (NCT01114139), researchers compared ferumoxytol injection to placebo.
The second trial (NCT01114204) was a comparison of ferumoxytol injection and iron sucrose.
In the third trial (NCT02694978), researchers compared ferumoxytol injection to ferric carboxymaltose injection (Injectafer®).
Details on these trials are included in the prescribing information for ferumoxytol injection, which is available at www.feraheme.com.
The prescribing information includes a boxed warning detailing the risk of fatal and serious hypersensitivity reactions, including anaphylaxis, in patients receiving ferumoxytol injection.
Ferumoxytol injection is a product of AMAG Pharmaceuticals, Inc.
The company has a patient access support program called AMAG Assist™. Uninsured or underinsured patients who need help paying for their ferumoxytol injection prescription can call 844-635-2624 to see if they qualify for help.
The US Food and Drug Administration (FDA) has expanded the approved indication for ferumoxytol injection (Feraheme®).
The drug is now approved to treat adults with iron deficiency anemia (IDA) who cannot tolerate or have had an unsatisfactory response to oral iron.
Ferumoxytol injection was previously approved by the FDA to treat IDA in adults with chronic kidney disease.
“Iron deficiency anemia is a serious and under-treated health condition which negatively impacts quality of life for millions of people, many of whom do not benefit from or cannot tolerate oral iron therapy,” said Michael Auerbach, MD, of Georgetown University School of Medicine in Washington, DC.
“Physicians now have a new option for patients who meet the broader ferumoxytol injection indication that can be administered in 15 minutes, providing a gram of iron in 2 doses as few as 3 days apart.”
The expanded approval for ferumoxytol injection was supported by a trio of phase 3 trials. All 3 trials included IDA patients who could not tolerate or had an unsatisfactory response to oral iron.
In the first trial (NCT01114139), researchers compared ferumoxytol injection to placebo.
The second trial (NCT01114204) was a comparison of ferumoxytol injection and iron sucrose.
In the third trial (NCT02694978), researchers compared ferumoxytol injection to ferric carboxymaltose injection (Injectafer®).
Details on these trials are included in the prescribing information for ferumoxytol injection, which is available at www.feraheme.com.
The prescribing information includes a boxed warning detailing the risk of fatal and serious hypersensitivity reactions, including anaphylaxis, in patients receiving ferumoxytol injection.
Ferumoxytol injection is a product of AMAG Pharmaceuticals, Inc.
The company has a patient access support program called AMAG Assist™. Uninsured or underinsured patients who need help paying for their ferumoxytol injection prescription can call 844-635-2624 to see if they qualify for help.
FDA approves ready-to-use bivalirudin
The US Food and Drug Administration (FDA) has approved a ready-to-use formulation of bivalirudin for use as an anticoagulant in patients undergoing percutaneous coronary intervention.
Baxter International Inc. expects to launch this frozen, premixed formulation of bivalirudin—Bivalirudin in 0.9% Sodium Chloride Injection—in the US early this year.
The product will be available in 2 commonly prescribed dosage forms and strengths: 250 mg of bivalirudin per 50 mL (5 mg/mL) and 500 mg of bivalirudin per 100 mL (5 mg/mL).
This frozen, premixed, ready-to-use bivalirudin makes use of Baxter’s proprietary frozen GALAXY container technology, a non-PVC and non-DEHP system specifically designed to create a ready-to-use format for unstable molecules.
Baxter’s premixed medications are manufactured to current Good Manufacturing Practice regulations established and monitored by the FDA.
The US Food and Drug Administration (FDA) has approved a ready-to-use formulation of bivalirudin for use as an anticoagulant in patients undergoing percutaneous coronary intervention.
Baxter International Inc. expects to launch this frozen, premixed formulation of bivalirudin—Bivalirudin in 0.9% Sodium Chloride Injection—in the US early this year.
The product will be available in 2 commonly prescribed dosage forms and strengths: 250 mg of bivalirudin per 50 mL (5 mg/mL) and 500 mg of bivalirudin per 100 mL (5 mg/mL).
This frozen, premixed, ready-to-use bivalirudin makes use of Baxter’s proprietary frozen GALAXY container technology, a non-PVC and non-DEHP system specifically designed to create a ready-to-use format for unstable molecules.
Baxter’s premixed medications are manufactured to current Good Manufacturing Practice regulations established and monitored by the FDA.
The US Food and Drug Administration (FDA) has approved a ready-to-use formulation of bivalirudin for use as an anticoagulant in patients undergoing percutaneous coronary intervention.
Baxter International Inc. expects to launch this frozen, premixed formulation of bivalirudin—Bivalirudin in 0.9% Sodium Chloride Injection—in the US early this year.
The product will be available in 2 commonly prescribed dosage forms and strengths: 250 mg of bivalirudin per 50 mL (5 mg/mL) and 500 mg of bivalirudin per 100 mL (5 mg/mL).
This frozen, premixed, ready-to-use bivalirudin makes use of Baxter’s proprietary frozen GALAXY container technology, a non-PVC and non-DEHP system specifically designed to create a ready-to-use format for unstable molecules.
Baxter’s premixed medications are manufactured to current Good Manufacturing Practice regulations established and monitored by the FDA.
EC authorizes brentuximab vedotin for CTCL
The European Commission (EC) has extended the conditional marketing authorization for brentuximab vedotin (Adcetris®).
The drug is now approved for use in adults with CD30-positive cutaneous T-cell lymphoma (CTCL) who have received at least 1 prior systemic therapy.
Brentuximab vedotin can be marketed for this indication in the member states of the European Union as well as in Norway, Liechtenstein, and Iceland.
Conditional marketing authorization from the EC is valid for 1 year and is reviewed annually.
With conditional authorization, drug developers are required to provide comprehensive data confirming a drug’s benefit-risk balance is positive. Once these data are available, a conditional marketing authorization may be converted to a standard marketing authorization.
Drugs are eligible for conditional marketing authorization if they are designated as orphan medicines, intended for use in emergency situations, or designed to treat, prevent, or diagnose seriously debilitating or life-threatening diseases.
The EC previously granted brentuximab vedotin conditional marketing authorization for the treatment of:
- Adults with CD30+ Hodgkin lymphoma who are at an increased risk of relapse or progression following autologous hematopoietic stem cell transplant (auto-HSCT)
- Adults with relapsed or refractory CD30+ Hodgkin lymphoma after auto-HSCT or after at least 2 prior therapies when auto-HSCT or multi-agent chemotherapy is not a treatment option
- Adults with relapsed or refractory systemic anaplastic large-cell lymphoma.
Brentuximab vedotin is under joint development by Seattle Genetics and Takeda Pharmaceutical Company Limited.
Phase 3 data
The EC’s latest authorization for brentuximab vedotin is based on data from the phase 3 ALCANZA trial.
Updated results from ALCANZA were presented at the 2017 ASH Annual Meeting in December. Results were previously presented at the 9th Annual T-cell Lymphoma Forum in January 2017 and published in The Lancet in June 2017.
The trial included 128 evaluable patients with CD30-positive CTCL who had received at least 1 prior systemic therapy.
Sixty-four patients were assigned to receive brentuximab vedotin, and 64 were assigned to receive the investigator’s choice of methotrexate or bexarotene (control arm). Patients received treatment for up to 1 year.
For the update, the median follow-up was 33.9 months.
There was a significant improvement in the rate of objective response lasting at least 4 months (ORR4) in the brentuximab vedotin arm compared to the control arm. The ORR4 was 60.9% and 7.8%, respectively (P<0.001). The complete response rate was 18.8% and 0%, respectively (P<0.001).
The median progression-free survival was 15.8 months in the brentuximab vedotin arm and 3.6 months in the control arm (hazard ratio=0.373; 95% CI, 0.245-0.569; P<0.001).
At time of analysis, 73% of patients in the brentuximab vedotin arm and 75% in the control arm had received 1 or more subsequent skin-directed or systemic therapies. The median time to next treatment was 14.2 months in the brentuximab vedotin arm and 6.1 months in the control arm (P<0.001).
Peripheral neuropathy was the most commonly reported adverse event in patients who received brentuximab vedotin. The incidence was 67% in these patients and 6% in controls.
In the brentuximab arm, 86% of patients reported resolution or improvement in peripheral neuropathy. Eighteen patients had ongoing peripheral neuropathy events, including 15 patients with grade 1 and 3 patients with grade 2 events.
The European Commission (EC) has extended the conditional marketing authorization for brentuximab vedotin (Adcetris®).
The drug is now approved for use in adults with CD30-positive cutaneous T-cell lymphoma (CTCL) who have received at least 1 prior systemic therapy.
Brentuximab vedotin can be marketed for this indication in the member states of the European Union as well as in Norway, Liechtenstein, and Iceland.
Conditional marketing authorization from the EC is valid for 1 year and is reviewed annually.
With conditional authorization, drug developers are required to provide comprehensive data confirming a drug’s benefit-risk balance is positive. Once these data are available, a conditional marketing authorization may be converted to a standard marketing authorization.
Drugs are eligible for conditional marketing authorization if they are designated as orphan medicines, intended for use in emergency situations, or designed to treat, prevent, or diagnose seriously debilitating or life-threatening diseases.
The EC previously granted brentuximab vedotin conditional marketing authorization for the treatment of:
- Adults with CD30+ Hodgkin lymphoma who are at an increased risk of relapse or progression following autologous hematopoietic stem cell transplant (auto-HSCT)
- Adults with relapsed or refractory CD30+ Hodgkin lymphoma after auto-HSCT or after at least 2 prior therapies when auto-HSCT or multi-agent chemotherapy is not a treatment option
- Adults with relapsed or refractory systemic anaplastic large-cell lymphoma.
Brentuximab vedotin is under joint development by Seattle Genetics and Takeda Pharmaceutical Company Limited.
Phase 3 data
The EC’s latest authorization for brentuximab vedotin is based on data from the phase 3 ALCANZA trial.
Updated results from ALCANZA were presented at the 2017 ASH Annual Meeting in December. Results were previously presented at the 9th Annual T-cell Lymphoma Forum in January 2017 and published in The Lancet in June 2017.
The trial included 128 evaluable patients with CD30-positive CTCL who had received at least 1 prior systemic therapy.
Sixty-four patients were assigned to receive brentuximab vedotin, and 64 were assigned to receive the investigator’s choice of methotrexate or bexarotene (control arm). Patients received treatment for up to 1 year.
For the update, the median follow-up was 33.9 months.
There was a significant improvement in the rate of objective response lasting at least 4 months (ORR4) in the brentuximab vedotin arm compared to the control arm. The ORR4 was 60.9% and 7.8%, respectively (P<0.001). The complete response rate was 18.8% and 0%, respectively (P<0.001).
The median progression-free survival was 15.8 months in the brentuximab vedotin arm and 3.6 months in the control arm (hazard ratio=0.373; 95% CI, 0.245-0.569; P<0.001).
At time of analysis, 73% of patients in the brentuximab vedotin arm and 75% in the control arm had received 1 or more subsequent skin-directed or systemic therapies. The median time to next treatment was 14.2 months in the brentuximab vedotin arm and 6.1 months in the control arm (P<0.001).
Peripheral neuropathy was the most commonly reported adverse event in patients who received brentuximab vedotin. The incidence was 67% in these patients and 6% in controls.
In the brentuximab arm, 86% of patients reported resolution or improvement in peripheral neuropathy. Eighteen patients had ongoing peripheral neuropathy events, including 15 patients with grade 1 and 3 patients with grade 2 events.
The European Commission (EC) has extended the conditional marketing authorization for brentuximab vedotin (Adcetris®).
The drug is now approved for use in adults with CD30-positive cutaneous T-cell lymphoma (CTCL) who have received at least 1 prior systemic therapy.
Brentuximab vedotin can be marketed for this indication in the member states of the European Union as well as in Norway, Liechtenstein, and Iceland.
Conditional marketing authorization from the EC is valid for 1 year and is reviewed annually.
With conditional authorization, drug developers are required to provide comprehensive data confirming a drug’s benefit-risk balance is positive. Once these data are available, a conditional marketing authorization may be converted to a standard marketing authorization.
Drugs are eligible for conditional marketing authorization if they are designated as orphan medicines, intended for use in emergency situations, or designed to treat, prevent, or diagnose seriously debilitating or life-threatening diseases.
The EC previously granted brentuximab vedotin conditional marketing authorization for the treatment of:
- Adults with CD30+ Hodgkin lymphoma who are at an increased risk of relapse or progression following autologous hematopoietic stem cell transplant (auto-HSCT)
- Adults with relapsed or refractory CD30+ Hodgkin lymphoma after auto-HSCT or after at least 2 prior therapies when auto-HSCT or multi-agent chemotherapy is not a treatment option
- Adults with relapsed or refractory systemic anaplastic large-cell lymphoma.
Brentuximab vedotin is under joint development by Seattle Genetics and Takeda Pharmaceutical Company Limited.
Phase 3 data
The EC’s latest authorization for brentuximab vedotin is based on data from the phase 3 ALCANZA trial.
Updated results from ALCANZA were presented at the 2017 ASH Annual Meeting in December. Results were previously presented at the 9th Annual T-cell Lymphoma Forum in January 2017 and published in The Lancet in June 2017.
The trial included 128 evaluable patients with CD30-positive CTCL who had received at least 1 prior systemic therapy.
Sixty-four patients were assigned to receive brentuximab vedotin, and 64 were assigned to receive the investigator’s choice of methotrexate or bexarotene (control arm). Patients received treatment for up to 1 year.
For the update, the median follow-up was 33.9 months.
There was a significant improvement in the rate of objective response lasting at least 4 months (ORR4) in the brentuximab vedotin arm compared to the control arm. The ORR4 was 60.9% and 7.8%, respectively (P<0.001). The complete response rate was 18.8% and 0%, respectively (P<0.001).
The median progression-free survival was 15.8 months in the brentuximab vedotin arm and 3.6 months in the control arm (hazard ratio=0.373; 95% CI, 0.245-0.569; P<0.001).
At time of analysis, 73% of patients in the brentuximab vedotin arm and 75% in the control arm had received 1 or more subsequent skin-directed or systemic therapies. The median time to next treatment was 14.2 months in the brentuximab vedotin arm and 6.1 months in the control arm (P<0.001).
Peripheral neuropathy was the most commonly reported adverse event in patients who received brentuximab vedotin. The incidence was 67% in these patients and 6% in controls.
In the brentuximab arm, 86% of patients reported resolution or improvement in peripheral neuropathy. Eighteen patients had ongoing peripheral neuropathy events, including 15 patients with grade 1 and 3 patients with grade 2 events.
Ofatumumab to be pulled from non-US markets
Novartis is planning to stop marketing ofatumumab (Arzerra®) outside the US, but the drug will still be available for certain patients.
The company plans to work with regulatory authorities to establish compassionate use programs for chronic lymphocytic leukemia (CLL) patients outside the US who are currently receiving ofatumumab.
Patients accessing these programs will be offered continued treatment with ofatumumab for as long as they benefit from it, free of charge.
And Novartis will continue to market ofatumumab for CLL in the US.
“Novartis’s intention to transition Arzerra to compassionate use programs in the non-US markets reflects the fact that many more drugs have become available for CLL over the last 5 years and that there is a low number of patients using Arzerra outside of the US market,” said Jan van de Winkel, PhD, chief executive officer of Genmab.
Ofatumumab is marketed under a collaboration agreement between Genmab and Novartis.
Because Novartis has decided to pull ofatumumab from non-US markets, the company will pay Genmab a lump sum of $50 million (USD) for lost potential milestones and royalties. Royalties will continue to be earned on net sales of the drug.
Novartis intends to start the transition to compassionate use programs as soon as the company and regulatory authorities have agreed to a plan.
The two phase 3 studies of ofatumumab in relapsing multiple sclerosis and the study in indolent non-Hodgkin lymphoma will not be affected by this change. All 3 trials will continue.
About ofatumumab
Ofatumumab is a monoclonal antibody designed to target CD20 on the surface of CLL cells and normal B lymphocytes.
In more than 60 countries worldwide, including the US and European Union member countries, ofatumumab is approved as monotherapy for CLL patients who are refractory to treatment with fludarabine and alemtuzumab.
Other approved indications for ofatumumab in the European Union include:
- In combination with chlorambucil or bendamustine to treat CLL patients who have not received prior therapy and are not eligible for fludarabine-based therapy
- In combination with fludarabine and cyclophosphamide to treat adults with relapsed CLL.
Other approved indications for ofatumumab in the US include:
- In combination with chlorambucil to treat previously untreated CLL patients for whom fludarabine-based therapy is considered inappropriate
- In combination with fludarabine and cyclophosphamide to treat patients with relapsed CLL
- For extended treatment of patients who are in complete or partial response after at least 2 lines of therapy for recurrent or progressive CLL.
Novartis is planning to stop marketing ofatumumab (Arzerra®) outside the US, but the drug will still be available for certain patients.
The company plans to work with regulatory authorities to establish compassionate use programs for chronic lymphocytic leukemia (CLL) patients outside the US who are currently receiving ofatumumab.
Patients accessing these programs will be offered continued treatment with ofatumumab for as long as they benefit from it, free of charge.
And Novartis will continue to market ofatumumab for CLL in the US.
“Novartis’s intention to transition Arzerra to compassionate use programs in the non-US markets reflects the fact that many more drugs have become available for CLL over the last 5 years and that there is a low number of patients using Arzerra outside of the US market,” said Jan van de Winkel, PhD, chief executive officer of Genmab.
Ofatumumab is marketed under a collaboration agreement between Genmab and Novartis.
Because Novartis has decided to pull ofatumumab from non-US markets, the company will pay Genmab a lump sum of $50 million (USD) for lost potential milestones and royalties. Royalties will continue to be earned on net sales of the drug.
Novartis intends to start the transition to compassionate use programs as soon as the company and regulatory authorities have agreed to a plan.
The two phase 3 studies of ofatumumab in relapsing multiple sclerosis and the study in indolent non-Hodgkin lymphoma will not be affected by this change. All 3 trials will continue.
About ofatumumab
Ofatumumab is a monoclonal antibody designed to target CD20 on the surface of CLL cells and normal B lymphocytes.
In more than 60 countries worldwide, including the US and European Union member countries, ofatumumab is approved as monotherapy for CLL patients who are refractory to treatment with fludarabine and alemtuzumab.
Other approved indications for ofatumumab in the European Union include:
- In combination with chlorambucil or bendamustine to treat CLL patients who have not received prior therapy and are not eligible for fludarabine-based therapy
- In combination with fludarabine and cyclophosphamide to treat adults with relapsed CLL.
Other approved indications for ofatumumab in the US include:
- In combination with chlorambucil to treat previously untreated CLL patients for whom fludarabine-based therapy is considered inappropriate
- In combination with fludarabine and cyclophosphamide to treat patients with relapsed CLL
- For extended treatment of patients who are in complete or partial response after at least 2 lines of therapy for recurrent or progressive CLL.
Novartis is planning to stop marketing ofatumumab (Arzerra®) outside the US, but the drug will still be available for certain patients.
The company plans to work with regulatory authorities to establish compassionate use programs for chronic lymphocytic leukemia (CLL) patients outside the US who are currently receiving ofatumumab.
Patients accessing these programs will be offered continued treatment with ofatumumab for as long as they benefit from it, free of charge.
And Novartis will continue to market ofatumumab for CLL in the US.
“Novartis’s intention to transition Arzerra to compassionate use programs in the non-US markets reflects the fact that many more drugs have become available for CLL over the last 5 years and that there is a low number of patients using Arzerra outside of the US market,” said Jan van de Winkel, PhD, chief executive officer of Genmab.
Ofatumumab is marketed under a collaboration agreement between Genmab and Novartis.
Because Novartis has decided to pull ofatumumab from non-US markets, the company will pay Genmab a lump sum of $50 million (USD) for lost potential milestones and royalties. Royalties will continue to be earned on net sales of the drug.
Novartis intends to start the transition to compassionate use programs as soon as the company and regulatory authorities have agreed to a plan.
The two phase 3 studies of ofatumumab in relapsing multiple sclerosis and the study in indolent non-Hodgkin lymphoma will not be affected by this change. All 3 trials will continue.
About ofatumumab
Ofatumumab is a monoclonal antibody designed to target CD20 on the surface of CLL cells and normal B lymphocytes.
In more than 60 countries worldwide, including the US and European Union member countries, ofatumumab is approved as monotherapy for CLL patients who are refractory to treatment with fludarabine and alemtuzumab.
Other approved indications for ofatumumab in the European Union include:
- In combination with chlorambucil or bendamustine to treat CLL patients who have not received prior therapy and are not eligible for fludarabine-based therapy
- In combination with fludarabine and cyclophosphamide to treat adults with relapsed CLL.
Other approved indications for ofatumumab in the US include:
- In combination with chlorambucil to treat previously untreated CLL patients for whom fludarabine-based therapy is considered inappropriate
- In combination with fludarabine and cyclophosphamide to treat patients with relapsed CLL
- For extended treatment of patients who are in complete or partial response after at least 2 lines of therapy for recurrent or progressive CLL.
Health systems plan to produce drugs themselves
A group of US health systems is planning to form a not-for-profit generic drug company with the goal of ending drug shortages and reducing prices for patients.
The company will either directly manufacture generic drugs or subcontract manufacturing to organizations it deems reputable.
“For people in the United States, there is a dangerous gap today between the demand and supply of affordable prescription drugs,” said Richard J. Gilfillan, MD, chief executive officer of Trinity Health, one of the health systems involved in this project.
“If the only way to provide our communities with affordable drugs is to produce them ourselves, then that is what we will do. We look forward to more healthcare systems around the country joining this people-centered effort.”
The organizations involved in this project include Intermountain Healthcare, Ascension, SSM Health, and Trinity Health, as well as the US Department of Veterans Affairs (although the department has not provided financial support for the project).
The 5 organizations represent more than 450 hospitals around the US, and other health systems are set to join the initiative as well.
“It’s an ambitious plan, but healthcare systems are in the best position to fix the problems in the generic drug market,” said Marc Harrison, MD, president and chief executive officer of Intermountain Healthcare.
“We witness, on a daily basis, how shortages of essential generic medications or egregious cost increases for those same drugs affect our patients. We are confident we can improve the situation for our patients by bringing much-needed competition to the generic drug market.”
The formation of this not-for-profit generic drug company will be guided by an advisory committee, which will include:
- Madhu Balachandran, retired executive vice-president of Global Operations, Amgen
- Don Berwick, MD, president emeritus and senior fellow, Institute for Healthcare Improvement; former Centers for Medicare & Medicaid Services administrator
- Clayton Christensen, professor at Harvard Business School and founder of Innosight
- Bob Kerrey, managing director, Allen & Company; former Nebraska governor and US senator
- Martin VanTrieste, retired senior vice-president and chief quality officer, Amgen
- Senior-level leaders from the organizations founding the company.
A group of US health systems is planning to form a not-for-profit generic drug company with the goal of ending drug shortages and reducing prices for patients.
The company will either directly manufacture generic drugs or subcontract manufacturing to organizations it deems reputable.
“For people in the United States, there is a dangerous gap today between the demand and supply of affordable prescription drugs,” said Richard J. Gilfillan, MD, chief executive officer of Trinity Health, one of the health systems involved in this project.
“If the only way to provide our communities with affordable drugs is to produce them ourselves, then that is what we will do. We look forward to more healthcare systems around the country joining this people-centered effort.”
The organizations involved in this project include Intermountain Healthcare, Ascension, SSM Health, and Trinity Health, as well as the US Department of Veterans Affairs (although the department has not provided financial support for the project).
The 5 organizations represent more than 450 hospitals around the US, and other health systems are set to join the initiative as well.
“It’s an ambitious plan, but healthcare systems are in the best position to fix the problems in the generic drug market,” said Marc Harrison, MD, president and chief executive officer of Intermountain Healthcare.
“We witness, on a daily basis, how shortages of essential generic medications or egregious cost increases for those same drugs affect our patients. We are confident we can improve the situation for our patients by bringing much-needed competition to the generic drug market.”
The formation of this not-for-profit generic drug company will be guided by an advisory committee, which will include:
- Madhu Balachandran, retired executive vice-president of Global Operations, Amgen
- Don Berwick, MD, president emeritus and senior fellow, Institute for Healthcare Improvement; former Centers for Medicare & Medicaid Services administrator
- Clayton Christensen, professor at Harvard Business School and founder of Innosight
- Bob Kerrey, managing director, Allen & Company; former Nebraska governor and US senator
- Martin VanTrieste, retired senior vice-president and chief quality officer, Amgen
- Senior-level leaders from the organizations founding the company.
A group of US health systems is planning to form a not-for-profit generic drug company with the goal of ending drug shortages and reducing prices for patients.
The company will either directly manufacture generic drugs or subcontract manufacturing to organizations it deems reputable.
“For people in the United States, there is a dangerous gap today between the demand and supply of affordable prescription drugs,” said Richard J. Gilfillan, MD, chief executive officer of Trinity Health, one of the health systems involved in this project.
“If the only way to provide our communities with affordable drugs is to produce them ourselves, then that is what we will do. We look forward to more healthcare systems around the country joining this people-centered effort.”
The organizations involved in this project include Intermountain Healthcare, Ascension, SSM Health, and Trinity Health, as well as the US Department of Veterans Affairs (although the department has not provided financial support for the project).
The 5 organizations represent more than 450 hospitals around the US, and other health systems are set to join the initiative as well.
“It’s an ambitious plan, but healthcare systems are in the best position to fix the problems in the generic drug market,” said Marc Harrison, MD, president and chief executive officer of Intermountain Healthcare.
“We witness, on a daily basis, how shortages of essential generic medications or egregious cost increases for those same drugs affect our patients. We are confident we can improve the situation for our patients by bringing much-needed competition to the generic drug market.”
The formation of this not-for-profit generic drug company will be guided by an advisory committee, which will include:
- Madhu Balachandran, retired executive vice-president of Global Operations, Amgen
- Don Berwick, MD, president emeritus and senior fellow, Institute for Healthcare Improvement; former Centers for Medicare & Medicaid Services administrator
- Clayton Christensen, professor at Harvard Business School and founder of Innosight
- Bob Kerrey, managing director, Allen & Company; former Nebraska governor and US senator
- Martin VanTrieste, retired senior vice-president and chief quality officer, Amgen
- Senior-level leaders from the organizations founding the company.
EC approves product to treat hemophilia A
The European Commission (EC) has granted marketing authorization for rurioctocog alfa pegol (Adynovi).
Rurioctocog alfa pegol (formerly BAX 855) is a pegylated, full-length, recombinant factor VIII product built on a previously licensed recombinant factor VIII product (Advate).
Rurioctocog alfa pegol is now EC-approved for on-demand and prophylactic use in patients age 12 and older with hemophilia A.
With the EC’s approval, Shire is authorized to market rurioctocog alfa pegol in all member states of the European Union as well as in Iceland, Liechtenstein, and Norway.
The marketing authorization for rurioctocog alfa pegol is based on outcomes from three phase 3 trials—a study of adolescents and adults, a trial of patients undergoing surgical procedures, and a study of pediatric patients.
Trial of adolescents/adults
This study included 137 patients, ages 12 to 58, with previously treated hemophilia A. They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with rurioctocog alfa pegol. One hundred and twenty-six patients completed the study.
Patients who received prophylaxis had a 90% reduction in annualized bleeding rate (ABR) compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
There were 7 adverse events (occurring in 6 patients) that were considered possibly related to rurioctocog alfa pegol. These included diarrhea, nausea, headache, and flushing.
These results were published in Blood in July 2015.
Perioperative study
This study included 15 patients with severe hemophilia A who were undergoing surgical procedures (11 of them major and 4 minor).
The patients received rurioctocog alfa pegol at varying doses and schedules, depending on each patient’s pharmacokinetic profile for major procedures or rurioctocog alfa pegol incremental recovery for minor procedures.
Intra-operative and peri-operative hemostatic efficacy of rurioctocog alfa pegol was deemed “excellent” for all 15 patients. The “excellent” rating meant that blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population.
Post-operatively (day 1 after the procedure), hemostatic efficacy was rated “good” for 1 procedure and “excellent” for the rest. The “good” rating meant that post-operative blood loss was up to 50% more than expected for the type of procedure performed in a non-hemophilic population.
There were no treatment-related adverse events or signs of immunogenicity in this trial.
Results were published in Haemophilia in June 2016.
Pediatric trial
This study included 66 patients, ages 1 to 11, who had previously treated hemophilia A. They received twice-weekly prophylaxis with rurioctocog alfa pegol (50 ± 10 IU/kg) for at least 6 months or 50 exposure days, whichever occurred last.
The median ABR was 2.0, and 38% of patients did not have any bleeding episodes.
None of the patients developed inhibitory antibodies, and there were no treatment-related adverse events.
Results from this trial were presented at the World Federation of Hemophilia 2016 World Congress in July 2016 and published in Haemophilia in November 2016.
The European Commission (EC) has granted marketing authorization for rurioctocog alfa pegol (Adynovi).
Rurioctocog alfa pegol (formerly BAX 855) is a pegylated, full-length, recombinant factor VIII product built on a previously licensed recombinant factor VIII product (Advate).
Rurioctocog alfa pegol is now EC-approved for on-demand and prophylactic use in patients age 12 and older with hemophilia A.
With the EC’s approval, Shire is authorized to market rurioctocog alfa pegol in all member states of the European Union as well as in Iceland, Liechtenstein, and Norway.
The marketing authorization for rurioctocog alfa pegol is based on outcomes from three phase 3 trials—a study of adolescents and adults, a trial of patients undergoing surgical procedures, and a study of pediatric patients.
Trial of adolescents/adults
This study included 137 patients, ages 12 to 58, with previously treated hemophilia A. They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with rurioctocog alfa pegol. One hundred and twenty-six patients completed the study.
Patients who received prophylaxis had a 90% reduction in annualized bleeding rate (ABR) compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
There were 7 adverse events (occurring in 6 patients) that were considered possibly related to rurioctocog alfa pegol. These included diarrhea, nausea, headache, and flushing.
These results were published in Blood in July 2015.
Perioperative study
This study included 15 patients with severe hemophilia A who were undergoing surgical procedures (11 of them major and 4 minor).
The patients received rurioctocog alfa pegol at varying doses and schedules, depending on each patient’s pharmacokinetic profile for major procedures or rurioctocog alfa pegol incremental recovery for minor procedures.
Intra-operative and peri-operative hemostatic efficacy of rurioctocog alfa pegol was deemed “excellent” for all 15 patients. The “excellent” rating meant that blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population.
Post-operatively (day 1 after the procedure), hemostatic efficacy was rated “good” for 1 procedure and “excellent” for the rest. The “good” rating meant that post-operative blood loss was up to 50% more than expected for the type of procedure performed in a non-hemophilic population.
There were no treatment-related adverse events or signs of immunogenicity in this trial.
Results were published in Haemophilia in June 2016.
Pediatric trial
This study included 66 patients, ages 1 to 11, who had previously treated hemophilia A. They received twice-weekly prophylaxis with rurioctocog alfa pegol (50 ± 10 IU/kg) for at least 6 months or 50 exposure days, whichever occurred last.
The median ABR was 2.0, and 38% of patients did not have any bleeding episodes.
None of the patients developed inhibitory antibodies, and there were no treatment-related adverse events.
Results from this trial were presented at the World Federation of Hemophilia 2016 World Congress in July 2016 and published in Haemophilia in November 2016.
The European Commission (EC) has granted marketing authorization for rurioctocog alfa pegol (Adynovi).
Rurioctocog alfa pegol (formerly BAX 855) is a pegylated, full-length, recombinant factor VIII product built on a previously licensed recombinant factor VIII product (Advate).
Rurioctocog alfa pegol is now EC-approved for on-demand and prophylactic use in patients age 12 and older with hemophilia A.
With the EC’s approval, Shire is authorized to market rurioctocog alfa pegol in all member states of the European Union as well as in Iceland, Liechtenstein, and Norway.
The marketing authorization for rurioctocog alfa pegol is based on outcomes from three phase 3 trials—a study of adolescents and adults, a trial of patients undergoing surgical procedures, and a study of pediatric patients.
Trial of adolescents/adults
This study included 137 patients, ages 12 to 58, with previously treated hemophilia A. They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with rurioctocog alfa pegol. One hundred and twenty-six patients completed the study.
Patients who received prophylaxis had a 90% reduction in annualized bleeding rate (ABR) compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
There were 7 adverse events (occurring in 6 patients) that were considered possibly related to rurioctocog alfa pegol. These included diarrhea, nausea, headache, and flushing.
These results were published in Blood in July 2015.
Perioperative study
This study included 15 patients with severe hemophilia A who were undergoing surgical procedures (11 of them major and 4 minor).
The patients received rurioctocog alfa pegol at varying doses and schedules, depending on each patient’s pharmacokinetic profile for major procedures or rurioctocog alfa pegol incremental recovery for minor procedures.
Intra-operative and peri-operative hemostatic efficacy of rurioctocog alfa pegol was deemed “excellent” for all 15 patients. The “excellent” rating meant that blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population.
Post-operatively (day 1 after the procedure), hemostatic efficacy was rated “good” for 1 procedure and “excellent” for the rest. The “good” rating meant that post-operative blood loss was up to 50% more than expected for the type of procedure performed in a non-hemophilic population.
There were no treatment-related adverse events or signs of immunogenicity in this trial.
Results were published in Haemophilia in June 2016.
Pediatric trial
This study included 66 patients, ages 1 to 11, who had previously treated hemophilia A. They received twice-weekly prophylaxis with rurioctocog alfa pegol (50 ± 10 IU/kg) for at least 6 months or 50 exposure days, whichever occurred last.
The median ABR was 2.0, and 38% of patients did not have any bleeding episodes.
None of the patients developed inhibitory antibodies, and there were no treatment-related adverse events.
Results from this trial were presented at the World Federation of Hemophilia 2016 World Congress in July 2016 and published in Haemophilia in November 2016.