Drugs

Dyne Files BLA for Duchenne Muscular Dystrophy Therapy

Dyne Therapeutics has submitted a BLA that seeks accelerated approval for its experimental Duchenne muscular dystrophy (DMD) therapy z-rostudirsen for patients amenable to exon 51 skipping.

The submission is based on data from the registrational expansion cohort of the Phase 1/2 DELIVER study, in which the therapy demonstrated a statistically significant increase in dystrophin production, the company says. Dyne also reports functional improvements across multiple clinical endpoints along with what it describes as a favorable safety profile.

The investigational therapy, also known as DYNE-251, is designed for patients with mutations in the DMD gene that can be treated through exon 51 skipping. Dyne is proposing a dosing regimen of 20 mg/kg administered intravenously once every four weeks.

The drug uses a phosphorodiamidate morpholino oligomer linked to an antibody fragment targeting transferrin receptor 1, a design intended to improve delivery into muscle tissue and the central nervous system, according to the company. The therapy is intended to restore production of near-full-length dystrophin, the protein missing in patients with DMD.

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