Drugs

Marathon Pharma Files NDA for Duchenne Drug

Marathon Pharmaceuticals has submitted an NDA for the investigational drug deflazacort for treating patients with Duchenne muscular dystrophy (DMD), the most common and most severe form of muscular dystrophy. The submission is supported by preclinical and clinical study data, including two pivotal clinical efficacy trials exclusively licensed by Marathon in more than 200 Duchenne patients aged five to 15 years.

“These data show that deflazacort improved muscle strength and other functional outcomes in patients with Duchenne regardless of genetic etiology and in one of the studies ambulation status,” the company says. Marathon additionally conducted seven clinical pharmacology and safety studies of deflazacort and nine preclinical studies to support either the initiation of clinical studies or marketing approval.

An expanded access program, Access DMD, is ongoing in the U.S. and provides deflazacort to patients with Duchenne free of charge during the NDA review process.

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