Drugs

Pyrukynd sNDA Extended Due to REMS Review

FDA has extended by three months its review of Agios Pharmaceuticals’ supplemental NDA seeking to expand the use of Pyrukynd (mitapivat) to adults with thalassemia. The extension, which was triggered by a Risk Evaluation and Mitigation Strategy (REMS) proposal that the company recently submitted, pushes back the user fee review action target date to 12/7, according to Agios. The REMS is intended to address potential risks of liver injury noted in the sNDA, it adds.

The application is supported by data from two Phase 3 studies: ENERGIZE, which tested the drug in adults with non-transfusion-dependent thalassemia, and ENERGIZE-T, which evaluated it in transfusion-dependent patients. Both trials were randomized, placebo-controlled, and conducted globally.

Thalassemia is described as a rare inherited blood disorder that disrupts hemoglobin production, leading to anemia, fatigue, and severe complications. Pyrukynd is currently approved in the U.S. for hemolytic anemia in adults with pyruvate kinase deficiency. It works by activating pyruvate kinase, a key enzyme in red blood cell metabolism, the company says.

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