Drugs

FDA Proposes Accelerated Approval Pathway for Stealth NDA

After extending its review and missing a 4/29 user fee review target date, FDA has now issued Stealth BioTherapeutics a complete response letter that proposes the company resubmit its elamipretide NDA to seek accelerated approval for treating Barth syndrome. The review was extended in January to allow FDA to review supplemental information provided in response to recent requests following a 10/10/2024 Cardiovascular and Renal Drugs Advisory Committee vote for approval.

The company says it submitted data in the NDA showing that knee extensor muscle strength, which improved by over 45% in its Phase 2 clinical trial, was significantly correlated with improvements on the six-minute walk test, an FDA-recognized indicator of clinical benefit. The NDA had proposed, and FDA has now agreed, to consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, it adds.

“We recognize that the FDA’s recommendation for an accelerated approval for elamipretide in Barth syndrome offers a path forward for this incurable pediatric disease that affects an incredibly small number of individuals worldwide,” the company says. “We hope the FDA will also prioritize ensuring rapid access for neonates affected by the disease subject to appropriate post-marketing safety monitoring. We are experiencing a sharp increase in emergency access requests for critically ill infants from medical experts worldwide following the recent publication of several positive case study reports.”

Stealth says the complete response letter also cited observations arising from a recent GMP surveillance inspection of a third-party manufacturing facility for elamipretide, although no specific deficiencies related to elamipretide were identified. It notes that the letter did not raise concerns with the clinical safety data, requesting only that a resubmission include any additional safety data collected since the original NDA submission. It plans to meet with FDA next month to discuss the proposed post-marketing study.

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