Drugs

FDA Lifts Hold on Myotonic Dystrophy Drug

FDA has lifted a partial clinical hold on Avidity Biosciences’ delpacibart etedesiran (del-desiran/AOC 1001) that is being evaluated for use in treating myotonic dystrophy Type 1. The investigational treatment is “designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies,” the company says.

FDA had placed the hold 9/2022 on all new participant enrollments due to a rare serious adverse event reported in a single participant in the 4 mg/kg cohort of the company’s Phase 1/2 MARINA trial. Last year, the agency “eased the partial clinical hold on AOC 1001, allowing Avidity to double the number of participants in the MARINA Open-Label Extension (MARINA-OLE) study receiving 4 mg/kg of AOC 1001,” the company says.

With the hold lifted, Avidity says it is beginning a global Phase 3 pivotal study (HARBOR) this quarter, with a primary endpoint of “video hand opening time (vHOT) and key secondary endpoints including muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and measured daily living activities.”

Delpacibart etedesiran works by reducing levels of a disease-related mRNA called DMPK, according to the company. It comprises a proprietary monoclonal antibody that binds to the transferrin receptor 1 conjugated with a siRNA targeting DMPK mRNA. In preclinical studies, the therapy successfully delivered siRNAs to muscle cells, “resulting in durable, dose-dependent reductions of DMPK RNA across a broad range of muscles including skeletal, cardiac, and smooth muscles,” 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