Drugs

Takeda Gains ‘Breakthrough’ for Pevonedistat

FDA has granted Takeda Pharmaceutical a breakthrough therapy designation for its investigational drug pevonedistat for treating patients with higher-risk myelodysplastic syndromes. The company says the candidate therapy, a first-in-class NEDD8-activating enzyme (NAE) inhibitor, could be the first novel therapy for the indication in more than a decade, expanding treatment options that have so far been limited to hypomethylating agent monotherapy alone. Even with current treatment options, outcomes for people living with HR-MDS remain poor.

The designation is based on the final analysis of the Pevonedistat-2001 Phase 2 study, which evaluated pevonedistat plus azacitidine versus azacitidine alone in patients with rare leukemias, including HR-MDS, the company says. “The FDA considered a number of endpoints, including overall survival, event-free survival, complete remission and transfusion independence, as well as the adverse event profile,” it says. In pre-clinical studies, the inhibition of NAE by pevonedistat blocked the modification of select proteins, which resulted in disruption of cell cycle progression and cell survival, leading to cancer cell death.” Additionally, pevonedistat in combination with azacitidine demonstrated “promising” clinical activity in a Phase 2 study of patients with higher-risk myelodysplastic syndromes, higher-risk chronic myelomonocytic leukemia and acute myeloid leukemia, Takeda adds.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome