Drugs

FDA Postpones Duchenne Drug Panel Meeting

FDA has postponed due to an expected snow storm a 1/22 Peripheral and Central Nervous System Advisory Committee meeting scheduled to review a Sarepta Therapeutics NDA for eteplirsen, indicated for treating Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A future meeting date will be announced in the Federal Register. The company has not been notified about any delay in the submission’s user fee review target date of 2/26.

In briefing materials released in advance of the meeting, FDA reviewers have raised concerns that data are not sufficient to allow the therapy’s approval. They said “data overall did not provide statistical evidence to support the efficacy in subjects who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping ... The only randomized controlled study submitted by the applicant, Study 201, can only be considered as exploratory because of study design and statistical analysis issues.”

FDA has been under pressure by patient advocates and Congress after it reversed course in 2013 and told Sarepta that an NDA for eteplirsen would be premature for treating DMD (see story). At the time, the agency cited recent developments that caused some alarm, including a failed study with a competitive product and recent natural history data in DMD, according to the company. The agency said the new data raise “considerable doubt” about “both the dystrophin biomarker and the supportive clinical efficacy assessed on the six-minute walk test (6MWT) in the Phase 2b clinical study of eteplirsen,” according to the company.

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