Drugs

Dynacure Gets Orphan Status for DYN101

FDA has granted Dynacure an orphan drug designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for treating centronuclear myopathies (CNM). The company expects to initiate a Phase 1/2 study (Unite-CNM) in a few months. The therapy is being developed in collaboration with Ionis Pharmaceuticals, which specializes in RNA-targeted drug discovery.

Centronuclear and myotubular myopathies are serious, rare, life-threatening disorders that affect skeletal muscles from birth, according to Dynacure. “The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered the link between an increase in DNM2 and the direct cause of the disease,” it says.

Preclinical studies have demonstrated that DYN101 has the potential to be disease modifying in CNM, with what the company calls "compelling" preclinical efficacy in treating animal models of X-linked recessive CNM and autosomal dominant CNM. “Prevention and reversion of the disease was observed with a clear dose-dependent improvement in whole body strength and mice survival,” Dynacure 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