User login
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.
Drug’s label updated to include risk of allergic reactions
The US Food and Drug Administration (FDA) and Tesaro, Inc., have updated the prescribing information for Varubi® (rolapitant) injectable emulsion to include a new warning about the risk of allergic reactions.
Varubi injectable emulsion is a substance P/neurokinin receptor antagonist approved to prevent delayed nausea and vomiting associated with chemotherapy in adults.
Since Varubi injectable emulsion gained FDA approval, there have been reports of anaphylaxis, anaphylactic shock, and other serious hypersensitivity reactions to the drug, some of which required hospitalization.
Now, the labeling for Varubi injectable emulsion has been changed to include information about these events. The changes include modifications to the CONTRAINDICATIONS, WARNINGS and PRECAUTIONS, and ADVERSE REACTIONS sections of the label.
Since Varubi injectable emulsion was introduced to the US market in late November 2017, at least 7000 doses of the drug have been administered to patients receiving emetogenic chemotherapy in the US, according to Tesaro.
Anaphylaxis, anaphylactic shock, and other serious hypersensitivity reactions have occurred during or soon after the infusion of Varubi. Most reactions have occurred within the first few minutes of administration.
The FDA has advised that patients who are hypersensitive to any component of Varubi injectable emulsion (including soybean oil) do not receive the drug. And patients with known allergies to legumes or other related allergens should be monitored closely.
The FDA said healthcare professionals should be vigilant for signs of hypersensitivity or anaphylaxis in all patients receiving Varubi injectable emulsion, both during administration and afterward.
Symptoms of anaphylaxis can include wheezing, difficulty breathing, swelling of the face or throat, hives, flushing, itching, abdominal cramping, abdominal pain, vomiting, back pain, chest pain, hypotension, and shock.
If anaphylaxis or any other serious hypersensitivity/infusion reaction occurs, Varubi injectable emulsion should be stopped immediately and permanently. The patient should receive appropriate medical management, including epinephrine and/or antihistamines.
To ensure patients and healthcare professionals are aware of the label update to Varubi injectable emulsion, Tesaro has issued a Dear Healthcare Professional letter. In addition, the updated prescribing information has been posted on the Varubi website.
For any questions about the use of Varubi injectable emulsion or to report adverse events related to the drug, contact Tesaro’s medical information department at 1-844-4-TESARO (1-844-483-7276).
Adverse events related to Varubi should also be reported to the FDA’s MedWatch Safety Information and Adverse Event Reporting Program.
The US Food and Drug Administration (FDA) and Tesaro, Inc., have updated the prescribing information for Varubi® (rolapitant) injectable emulsion to include a new warning about the risk of allergic reactions.
Varubi injectable emulsion is a substance P/neurokinin receptor antagonist approved to prevent delayed nausea and vomiting associated with chemotherapy in adults.
Since Varubi injectable emulsion gained FDA approval, there have been reports of anaphylaxis, anaphylactic shock, and other serious hypersensitivity reactions to the drug, some of which required hospitalization.
Now, the labeling for Varubi injectable emulsion has been changed to include information about these events. The changes include modifications to the CONTRAINDICATIONS, WARNINGS and PRECAUTIONS, and ADVERSE REACTIONS sections of the label.
Since Varubi injectable emulsion was introduced to the US market in late November 2017, at least 7000 doses of the drug have been administered to patients receiving emetogenic chemotherapy in the US, according to Tesaro.
Anaphylaxis, anaphylactic shock, and other serious hypersensitivity reactions have occurred during or soon after the infusion of Varubi. Most reactions have occurred within the first few minutes of administration.
The FDA has advised that patients who are hypersensitive to any component of Varubi injectable emulsion (including soybean oil) do not receive the drug. And patients with known allergies to legumes or other related allergens should be monitored closely.
The FDA said healthcare professionals should be vigilant for signs of hypersensitivity or anaphylaxis in all patients receiving Varubi injectable emulsion, both during administration and afterward.
Symptoms of anaphylaxis can include wheezing, difficulty breathing, swelling of the face or throat, hives, flushing, itching, abdominal cramping, abdominal pain, vomiting, back pain, chest pain, hypotension, and shock.
If anaphylaxis or any other serious hypersensitivity/infusion reaction occurs, Varubi injectable emulsion should be stopped immediately and permanently. The patient should receive appropriate medical management, including epinephrine and/or antihistamines.
To ensure patients and healthcare professionals are aware of the label update to Varubi injectable emulsion, Tesaro has issued a Dear Healthcare Professional letter. In addition, the updated prescribing information has been posted on the Varubi website.
For any questions about the use of Varubi injectable emulsion or to report adverse events related to the drug, contact Tesaro’s medical information department at 1-844-4-TESARO (1-844-483-7276).
Adverse events related to Varubi should also be reported to the FDA’s MedWatch Safety Information and Adverse Event Reporting Program.
The US Food and Drug Administration (FDA) and Tesaro, Inc., have updated the prescribing information for Varubi® (rolapitant) injectable emulsion to include a new warning about the risk of allergic reactions.
Varubi injectable emulsion is a substance P/neurokinin receptor antagonist approved to prevent delayed nausea and vomiting associated with chemotherapy in adults.
Since Varubi injectable emulsion gained FDA approval, there have been reports of anaphylaxis, anaphylactic shock, and other serious hypersensitivity reactions to the drug, some of which required hospitalization.
Now, the labeling for Varubi injectable emulsion has been changed to include information about these events. The changes include modifications to the CONTRAINDICATIONS, WARNINGS and PRECAUTIONS, and ADVERSE REACTIONS sections of the label.
Since Varubi injectable emulsion was introduced to the US market in late November 2017, at least 7000 doses of the drug have been administered to patients receiving emetogenic chemotherapy in the US, according to Tesaro.
Anaphylaxis, anaphylactic shock, and other serious hypersensitivity reactions have occurred during or soon after the infusion of Varubi. Most reactions have occurred within the first few minutes of administration.
The FDA has advised that patients who are hypersensitive to any component of Varubi injectable emulsion (including soybean oil) do not receive the drug. And patients with known allergies to legumes or other related allergens should be monitored closely.
The FDA said healthcare professionals should be vigilant for signs of hypersensitivity or anaphylaxis in all patients receiving Varubi injectable emulsion, both during administration and afterward.
Symptoms of anaphylaxis can include wheezing, difficulty breathing, swelling of the face or throat, hives, flushing, itching, abdominal cramping, abdominal pain, vomiting, back pain, chest pain, hypotension, and shock.
If anaphylaxis or any other serious hypersensitivity/infusion reaction occurs, Varubi injectable emulsion should be stopped immediately and permanently. The patient should receive appropriate medical management, including epinephrine and/or antihistamines.
To ensure patients and healthcare professionals are aware of the label update to Varubi injectable emulsion, Tesaro has issued a Dear Healthcare Professional letter. In addition, the updated prescribing information has been posted on the Varubi website.
For any questions about the use of Varubi injectable emulsion or to report adverse events related to the drug, contact Tesaro’s medical information department at 1-844-4-TESARO (1-844-483-7276).
Adverse events related to Varubi should also be reported to the FDA’s MedWatch Safety Information and Adverse Event Reporting Program.
FDA expands approval for arsenic trioxide
The US Food and Drug Administration (FDA) has expanded the approved use of arsenic trioxide (TRISENOX®) injection.
The drug is now approved for use in combination with all-trans retinoic acid (ATRA) for the treatment of adults with newly diagnosed, low-risk acute promyelocytic leukemia (APL) whose disease is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.
Arsenic trioxide is also FDA-approved for induction of remission and consolidation in patients with APL who are refractory to, or have relapsed after, retinoid and anthracycline chemotherapy and whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.
“This label expansion represents an important benefit, as TRISENOX is now an FDA-approved, first-line treatment option for patients with acute promyelocytic leukemia,” said Paul Rittman, senior vice-president and general manager of Teva Oncology.
The expanded approval for arsenic trioxide was based on a priority review by the FDA of data from the scientific literature and a review of Teva’s global safety database for arsenic trioxide.
Data from this database were presented at the 2016 ASH Annual Meeting.
According to the presentation, the most common adverse events observed in patients receiving arsenic trioxide were QT prolongation, decrease in white blood cells, APL differentiation syndrome, febrile neutropenia, neutropenia, pyrexia, alanine aminotransferase increase, neutrophil decrease, platelet count decrease, aspartate aminotransferase increase, leukocytosis, and pancytopenia.
The combination of arsenic trioxide and ATRA was evaluated in a phase 3 trial of patients with APL. Results from this trial were published in the Journal of Clinical Oncology in February 2017.
The study included 276 adults (ages 18 to 71) with newly diagnosed, low- or intermediate-risk APL. Patients were randomized to receive ATRA plus arsenic trioxide or ATRA plus chemotherapy.
A total of 263 patients were evaluable for response to induction. One hundred percent of patients in the arsenic trioxide arm (127/127) achieved a complete response (CR), as did 97% (132/136) of patients in the chemotherapy arm (P=0.12).
After a median follow-up of 40.6 months, the event-free survival was 97.3% in the arsenic trioxide arm and 80% in the chemotherapy arm (P<0.001). The cumulative incidence of relapse was 1.9% and 13.9%, respectively (P=0.0013).
At 50 months, the overall survival was 99.2% in the arsenic trioxide arm and 92.6% in the chemotherapy arm (P=0.0073).
After induction, there were 2 relapses and 1 death in CR in the arsenic trioxide arm.
In the chemotherapy arm, there were 2 instances of molecular resistance after third consolidation, 15 relapses, 5 deaths in CR, and 2 patients who developed a therapy-related myeloid neoplasm.
The US Food and Drug Administration (FDA) has expanded the approved use of arsenic trioxide (TRISENOX®) injection.
The drug is now approved for use in combination with all-trans retinoic acid (ATRA) for the treatment of adults with newly diagnosed, low-risk acute promyelocytic leukemia (APL) whose disease is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.
Arsenic trioxide is also FDA-approved for induction of remission and consolidation in patients with APL who are refractory to, or have relapsed after, retinoid and anthracycline chemotherapy and whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.
“This label expansion represents an important benefit, as TRISENOX is now an FDA-approved, first-line treatment option for patients with acute promyelocytic leukemia,” said Paul Rittman, senior vice-president and general manager of Teva Oncology.
The expanded approval for arsenic trioxide was based on a priority review by the FDA of data from the scientific literature and a review of Teva’s global safety database for arsenic trioxide.
Data from this database were presented at the 2016 ASH Annual Meeting.
According to the presentation, the most common adverse events observed in patients receiving arsenic trioxide were QT prolongation, decrease in white blood cells, APL differentiation syndrome, febrile neutropenia, neutropenia, pyrexia, alanine aminotransferase increase, neutrophil decrease, platelet count decrease, aspartate aminotransferase increase, leukocytosis, and pancytopenia.
The combination of arsenic trioxide and ATRA was evaluated in a phase 3 trial of patients with APL. Results from this trial were published in the Journal of Clinical Oncology in February 2017.
The study included 276 adults (ages 18 to 71) with newly diagnosed, low- or intermediate-risk APL. Patients were randomized to receive ATRA plus arsenic trioxide or ATRA plus chemotherapy.
A total of 263 patients were evaluable for response to induction. One hundred percent of patients in the arsenic trioxide arm (127/127) achieved a complete response (CR), as did 97% (132/136) of patients in the chemotherapy arm (P=0.12).
After a median follow-up of 40.6 months, the event-free survival was 97.3% in the arsenic trioxide arm and 80% in the chemotherapy arm (P<0.001). The cumulative incidence of relapse was 1.9% and 13.9%, respectively (P=0.0013).
At 50 months, the overall survival was 99.2% in the arsenic trioxide arm and 92.6% in the chemotherapy arm (P=0.0073).
After induction, there were 2 relapses and 1 death in CR in the arsenic trioxide arm.
In the chemotherapy arm, there were 2 instances of molecular resistance after third consolidation, 15 relapses, 5 deaths in CR, and 2 patients who developed a therapy-related myeloid neoplasm.
The US Food and Drug Administration (FDA) has expanded the approved use of arsenic trioxide (TRISENOX®) injection.
The drug is now approved for use in combination with all-trans retinoic acid (ATRA) for the treatment of adults with newly diagnosed, low-risk acute promyelocytic leukemia (APL) whose disease is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.
Arsenic trioxide is also FDA-approved for induction of remission and consolidation in patients with APL who are refractory to, or have relapsed after, retinoid and anthracycline chemotherapy and whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.
“This label expansion represents an important benefit, as TRISENOX is now an FDA-approved, first-line treatment option for patients with acute promyelocytic leukemia,” said Paul Rittman, senior vice-president and general manager of Teva Oncology.
The expanded approval for arsenic trioxide was based on a priority review by the FDA of data from the scientific literature and a review of Teva’s global safety database for arsenic trioxide.
Data from this database were presented at the 2016 ASH Annual Meeting.
According to the presentation, the most common adverse events observed in patients receiving arsenic trioxide were QT prolongation, decrease in white blood cells, APL differentiation syndrome, febrile neutropenia, neutropenia, pyrexia, alanine aminotransferase increase, neutrophil decrease, platelet count decrease, aspartate aminotransferase increase, leukocytosis, and pancytopenia.
The combination of arsenic trioxide and ATRA was evaluated in a phase 3 trial of patients with APL. Results from this trial were published in the Journal of Clinical Oncology in February 2017.
The study included 276 adults (ages 18 to 71) with newly diagnosed, low- or intermediate-risk APL. Patients were randomized to receive ATRA plus arsenic trioxide or ATRA plus chemotherapy.
A total of 263 patients were evaluable for response to induction. One hundred percent of patients in the arsenic trioxide arm (127/127) achieved a complete response (CR), as did 97% (132/136) of patients in the chemotherapy arm (P=0.12).
After a median follow-up of 40.6 months, the event-free survival was 97.3% in the arsenic trioxide arm and 80% in the chemotherapy arm (P<0.001). The cumulative incidence of relapse was 1.9% and 13.9%, respectively (P=0.0013).
At 50 months, the overall survival was 99.2% in the arsenic trioxide arm and 92.6% in the chemotherapy arm (P=0.0073).
After induction, there were 2 relapses and 1 death in CR in the arsenic trioxide arm.
In the chemotherapy arm, there were 2 instances of molecular resistance after third consolidation, 15 relapses, 5 deaths in CR, and 2 patients who developed a therapy-related myeloid neoplasm.
Emicizumab still available despite legal issues
The Roche Group has issued a statement reassuring the US hemophilia community that legal issues are not affecting patient access to emicizumab (Hemlibra), at least for the time being.
Emicizumab is a bispecific factor IXa- and factor X-directed antibody approved in the US as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A and factor VIII (FVIII) inhibitors.
The Roche Group—which consists of Genentech in the US, Chugai in Japan, and Roche in the rest of the world—said emicizumab is still available for these patients, despite a legal battle with Baxalta, a wholly owned subsidiary of Shire.
In May 2017, Baxalta sued Genentech and Chugai for allegedly infringing upon US Patent No. 7,033,590.
The patent covers factor IX/factor IXa antibodies and antibody derivatives. It was issued to Baxter in April 2006 and assigned to Baxalta in March 2016. The patent is still active.
According to Baxalta, Genentech and Chugai are infringing on the patent by manufacturing, selling, or importing emicizumab. Therefore, Baxalta is seeking judgment in its favor and monetary damages.
The trial for this case is scheduled for September 2019.
However, in December 2017, Baxalta filed a motion for a preliminary injunction that would prevent the sale of emicizumab in the US. If granted, the injunction would prevent the following patients from receiving emicizumab:
- Hemophilia A patients with inhibitors (an inhibitor titer of greater than 5 Bethesda units) who cannot be treated effectively with FVIII replacement therapy, unless (i) they have already started emicizumab before the injunction is granted or (ii) they have previous experience with on-demand or prophylactic bypassing agents and their needs are not being met, as defined by Shire using criteria that include experiencing certain life- or limb-threatening bleeds or venous access issues.
- Hemophilia A patients who have an inhibitor titer less than or equal to 5 Bethesda units or who can be effectively treated with FVIII replacement therapy, regardless of whether they have already started emicizumab.
- Hemophilia A patients without inhibitors, regardless of whether they have already started emicizumab.
The Roche Group said it believes Baxalta’s claim is not valid, emicizumab does not infringe upon the patent, and the group will oppose the injunction.
The court’s decision on the injunction is expected this summer. In the meantime, emicizumab is still available for the aforementioned patients.
The Roche Group has issued a statement reassuring the US hemophilia community that legal issues are not affecting patient access to emicizumab (Hemlibra), at least for the time being.
Emicizumab is a bispecific factor IXa- and factor X-directed antibody approved in the US as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A and factor VIII (FVIII) inhibitors.
The Roche Group—which consists of Genentech in the US, Chugai in Japan, and Roche in the rest of the world—said emicizumab is still available for these patients, despite a legal battle with Baxalta, a wholly owned subsidiary of Shire.
In May 2017, Baxalta sued Genentech and Chugai for allegedly infringing upon US Patent No. 7,033,590.
The patent covers factor IX/factor IXa antibodies and antibody derivatives. It was issued to Baxter in April 2006 and assigned to Baxalta in March 2016. The patent is still active.
According to Baxalta, Genentech and Chugai are infringing on the patent by manufacturing, selling, or importing emicizumab. Therefore, Baxalta is seeking judgment in its favor and monetary damages.
The trial for this case is scheduled for September 2019.
However, in December 2017, Baxalta filed a motion for a preliminary injunction that would prevent the sale of emicizumab in the US. If granted, the injunction would prevent the following patients from receiving emicizumab:
- Hemophilia A patients with inhibitors (an inhibitor titer of greater than 5 Bethesda units) who cannot be treated effectively with FVIII replacement therapy, unless (i) they have already started emicizumab before the injunction is granted or (ii) they have previous experience with on-demand or prophylactic bypassing agents and their needs are not being met, as defined by Shire using criteria that include experiencing certain life- or limb-threatening bleeds or venous access issues.
- Hemophilia A patients who have an inhibitor titer less than or equal to 5 Bethesda units or who can be effectively treated with FVIII replacement therapy, regardless of whether they have already started emicizumab.
- Hemophilia A patients without inhibitors, regardless of whether they have already started emicizumab.
The Roche Group said it believes Baxalta’s claim is not valid, emicizumab does not infringe upon the patent, and the group will oppose the injunction.
The court’s decision on the injunction is expected this summer. In the meantime, emicizumab is still available for the aforementioned patients.
The Roche Group has issued a statement reassuring the US hemophilia community that legal issues are not affecting patient access to emicizumab (Hemlibra), at least for the time being.
Emicizumab is a bispecific factor IXa- and factor X-directed antibody approved in the US as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A and factor VIII (FVIII) inhibitors.
The Roche Group—which consists of Genentech in the US, Chugai in Japan, and Roche in the rest of the world—said emicizumab is still available for these patients, despite a legal battle with Baxalta, a wholly owned subsidiary of Shire.
In May 2017, Baxalta sued Genentech and Chugai for allegedly infringing upon US Patent No. 7,033,590.
The patent covers factor IX/factor IXa antibodies and antibody derivatives. It was issued to Baxter in April 2006 and assigned to Baxalta in March 2016. The patent is still active.
According to Baxalta, Genentech and Chugai are infringing on the patent by manufacturing, selling, or importing emicizumab. Therefore, Baxalta is seeking judgment in its favor and monetary damages.
The trial for this case is scheduled for September 2019.
However, in December 2017, Baxalta filed a motion for a preliminary injunction that would prevent the sale of emicizumab in the US. If granted, the injunction would prevent the following patients from receiving emicizumab:
- Hemophilia A patients with inhibitors (an inhibitor titer of greater than 5 Bethesda units) who cannot be treated effectively with FVIII replacement therapy, unless (i) they have already started emicizumab before the injunction is granted or (ii) they have previous experience with on-demand or prophylactic bypassing agents and their needs are not being met, as defined by Shire using criteria that include experiencing certain life- or limb-threatening bleeds or venous access issues.
- Hemophilia A patients who have an inhibitor titer less than or equal to 5 Bethesda units or who can be effectively treated with FVIII replacement therapy, regardless of whether they have already started emicizumab.
- Hemophilia A patients without inhibitors, regardless of whether they have already started emicizumab.
The Roche Group said it believes Baxalta’s claim is not valid, emicizumab does not infringe upon the patent, and the group will oppose the injunction.
The court’s decision on the injunction is expected this summer. In the meantime, emicizumab is still available for the aforementioned patients.
Generic bortezomib available in US
Fresenius Kabi has introduced its generic version of Velcade, Bortezomib for Injection, to the US market.
This is the first intravenous alternative to Velcade available in the US.
Bortezomib for Injection is available as a single dose vial containing 3.5 mg of lyophilized powder.
The product is approved to treat patients with multiple myeloma and patients with mantle cell lymphoma who have received at least 1 prior therapy.
For details, see the prescribing information for Bortezomib for Injection.
Velcade is a registered trademark of Millennium Pharmaceuticals, Inc.
Fresenius Kabi has introduced its generic version of Velcade, Bortezomib for Injection, to the US market.
This is the first intravenous alternative to Velcade available in the US.
Bortezomib for Injection is available as a single dose vial containing 3.5 mg of lyophilized powder.
The product is approved to treat patients with multiple myeloma and patients with mantle cell lymphoma who have received at least 1 prior therapy.
For details, see the prescribing information for Bortezomib for Injection.
Velcade is a registered trademark of Millennium Pharmaceuticals, Inc.
Fresenius Kabi has introduced its generic version of Velcade, Bortezomib for Injection, to the US market.
This is the first intravenous alternative to Velcade available in the US.
Bortezomib for Injection is available as a single dose vial containing 3.5 mg of lyophilized powder.
The product is approved to treat patients with multiple myeloma and patients with mantle cell lymphoma who have received at least 1 prior therapy.
For details, see the prescribing information for Bortezomib for Injection.
Velcade is a registered trademark of Millennium Pharmaceuticals, Inc.