Drugs

FDA Holds Up Sarepta Gene Therapy Trial

FDA’s nervousness with gene therapies continues as it holds up a Sarepta Therapeutics clinical trial by asking for an additional potency assay to release SRP-9001 commercial process material prior to dosing in the planned Phase 3 trial. SRP-9001 is the company’s investigational gene transfer therapy for treating Duchenne muscular dystrophy. Financial analysts are saying this is a few months delay in beginning the trial, which may give Pfizer a boost in moving its Duchenne gene therapy, PF-06939926, into Phase 3, which is planned for later this year.

To date, FDA has approved four gene therapy products and it has been exercising caution as it navigates regulatory oversight of the nascent therapeutic field. Last month, the agency issued a complete response letter to BioMarin Pharmaceutical for its valoctocogene roxaparvovec gene therapy for severe hemophilia A. The company said the agency is now requesting two years of data from an ongoing Phase 3 study to provide evidence of a durable effect using annualized bleeding rate as the primary endpoint. BioMarin says the last patient enrolled in the study will complete two years of follow-up in 11/2021.

Earlier this year, FDA issued six final guidances on gene therapy manufacturing and clinical development of products plus a draft guidance on interpreting the sameness of gene therapy products under the orphan drug regulations (see earlier story). “The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality,” the agency said at the time. “In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often can’t be fully answered in pre-market trials of reasonable size and duration. For some gene therapy products, therefore, although they have met the FDA’s standards for approval, we may need to accept some level of uncertainty around questions of the duration of the response at the time of marketing authorization. Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.”

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