Drugs

Orphan Drug Status for Tivorsan Duchenne Drug

FDA has granted Tivorsan Pharmaceuticals an orphan drug designation for TVN-102 (biglycan) and its use in treating Duchenne Muscular Dystrophy. The company says it is on track to submit an IND and begin clinical trials in 2017.

TVN-102 is described by Tivorsan as a small, naturally occurring extracellular matrix protein found in muscle. “It has a unique mechanism of action, that of upregulating utrophin, nNOS (neuronal nitric oxide synthase) and other dystrophin-associated proteins in the muscle membrane, as well as controlling MuSK (muscle specific kinase) activity and localization at the nerve-muscle synapse,” it says. “Tests in the most widely used mouse model of DMD have shown that systemically delivered TVN-102 upregulates utrophin and activates the dystrophin-independent membrane stabilization program. TVN-102 has been shown to improve muscle health and function in a mouse model. Mice treated with TVN-102 also show reduced muscle cell degeneration and death.”

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