Drugs

Audentes Therapeutics IND Cleared by FDA

FDA has approved an Audentes Therapeutics IND for AT132, the company’s gene therapy product candidate to treat X-Linked Myotubular Myopathy (XLMTM). The IND covers the company’s plans to begin ASPIRO, a multicenter, multinational, open-label, ascending dose Phase 1/2 clinical study of AT132. Preliminary data from ASPIRO are expected to be available in the fourth quarter.

In addition to ASPIRO, the therapy’s clinical development program includes RECENSUS, a retrospective medical chart review, for which Audentes recently announced data from an initial analysis of 112 male subjects. “This analysis confirmed and expanded upon the understanding of the medical burden of XLMTM on patients, families and the healthcare system,” Audentes says.

XLMTM is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age. Caused by mutations in the MTM1 gene, XLMTM encodes a protein called myotubularinm, which plays an important role in the development, maintenance and function of skeletal muscle cells. AT132 is made up of an AAV8 vector containing a functional copy of the MTM1 gene. “Multiple studies in animal models of XLMTM have demonstrated that a single administration of AT132 improved disease symptoms and survival rates, with no significant AT132-related adverse events or safety findings,” the company 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