Biologics

Public Citizen Urges FDA to Reject Duchenne BLA

Public Citizen’s Health Research Group is urging FDA to not grant accelerated approval to Sarepta Therapeutics’ gene therapy SRP-9001 for treating ambulatory Duchenne muscular dystrophy (DMD) because the data do not justify approval. In a 6/15 letter to the agency, the advocacy group says the data failed to demonstrate significant muscle-function sparing in the only randomized clinical trial so far. “Moreover, the treatment has safety concerns — most notably that the viral vector needed to deliver this gene therapy cannot be used repeatedly, even for another treatment that might later prove to be safe and effective,” it says. “If this potential treatment for DMD is fast-tracked with accelerated approval, other companies may be encouraged to rush unconfirmed gene therapies to the marketplace.”

Public Citizen’s letter said the group was concerned about the anecdotal video evidence of patients’ improvements presented in last month’s advisory committee meeting (see earlier story). FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee 5/12 voted 8 to 6 to recommend approval of the Sarepta BLA. A briefing document issued in advance of the meeting said agency medical reviewers found that “the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with DMD.”

In voting no at the meeting, Johns Hopkins University professor Caleb Alexander agreed that patient videos of progress on therapy were “compelling,” but he said accelerated approval needs more than that. Alexander said this therapy is not an instance where a single-arm, open label clinical study and external controls may suffice. “So, I think the totality of evidence and what we reviewed in the briefing simply doesn't rise to the threshold of substantial evidence that's required for accelerated approval.”

A final FDA decision on the application is expected by 6/22.

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

Read more

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