Drugs

Clinical Hold on Gene Therapy Lifted

FDA has lifted a clinical hold against Solid Biosciences’ IGNITE DMD Phase 1/2 clinical trial involving SGT-001, a novel adeno-associated viral vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy. The hold was originally placed 11/2019 after the company reported a serious adverse event that occurred in the sixth patient dosed in the trial. In April, Solid says it provided FDA with information and measures intended to improve patient safety and in May, Solid received written communication that the trial remained on hold. In June, the company submitted a response that provided data on manufacturing process improvements. FDA next responded by maintaining the clinical hold and requesting further manufacturing information, updated safety and efficacy data for all patients dosed, and providing direction on total viral load to be administered per patient. Based on the company’s response to these requests, Solid Bio says FDA has acknowledged that all clinical hold questions have been satisfactorily addressed.

SGT-001 is described by the company as a novel adeno-associated viral vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy. It is designed to deliver a synthetic dystrophin gene called microdystrophin to the body. “This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase,” Solid says. “Data from Solid’s preclinical program suggest that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.”

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