Drugs

Blueprint Medicine’s Mastocytosis NDA Approved

FDA has approved a Blueprint Medicines NDA for Ayvakit (avapritinib) for treating adults with indolent systemic mastocytosis (ISM). The rare hematologic disorder can lead to several debilitating symptoms across multiple organ systems, according to the company. The drug was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease, it says.

Approval was based on data from the double-blind, placebo-controlled PIONEER trial that evaluated Ayvakit 25 mg once daily plus best supportive care or placebo plus best supportive care. “Ayvakit demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden,” Blueprint says.

The company says most adverse reactions in the trial were mild to moderate in severity. The most common were eye edema, dizziness, peripheral edema and flushing. “Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1% of patients,” it 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