Drugs

Sarepta Scraps Next-Gen Duchenne Drug

Sarepta says it is discontinuing the development of SRP-5051 (vesleteplirsen), an investigational, next-generation peptide conjugated phosphorodiamidate morpholino oligomer for treating patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. The decision was based on FDA feedback about the therapy’s risk-benefit, according to the company, particularly reports of abnormally low magnesium (hypomagnesemia) that continued to be reported in patients taking the therapy.

Earlier this year, Sarepta reported positive data from Part B of the Phase 2 MOMENTUM study, which assessed vesleteplirsen in patients aged 8 to 21 years who are amenable to exon 51 skipping. The company noted at the time that cases of hypomagnesemia had previously been identified and that this was “managed and monitored” during the trial.

During an 11/6 earnings call, company executives noted that the cases continued to be seen including in some patients who had discontinued treatment. The company said it was also told by FDA that the accelerated approval pathway was not a viable option for vesleteplirsen based on the current data.

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