Drugs

Dyne Gets Breakthrough Status for Duchenne Therapy

FDA has granted Dyne Therapeutics a breakthrough therapy designation for DYNE-251 for treating patients with Duchenne muscular dystrophy (DMD) that is amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. “As we’ve previously disclosed, DYNE-251 has demonstrated sustained functional improvement through 18 months, as assessed by key measures such as time to rise and stride velocity 95th centile,” the company says.

Dyne has completed enrollment of 32 patients in the DELIVER trial’s “registrational expansion cohort,” with data from this cohort expected before year end. Dyne expects submit a BLA submission seeking accelerated approval in early 2026.

DYNE-251 is described by the company as a phosphorodiamidate morpholino oligomer conjugated to an antigen-binding fragment that binds to the transferrin receptor 1. “It is designed to enable the production of near full-length dystrophin in muscle and the central nervous system (CNS) to provide functional improvement,” it says.

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