Drugs

FDA, Rocket Agree on Phase 2 Danon Disease Trial

FDA and Rocket Pharmaceuticals have reached an agreement on a pivotal Phase 2 trial of the company’s RP-A501 to treat Danon Disease. Rocket CEO Gaurav Shah says the investigational gene therapy infusion “has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other treatment options.”

RP-A501 is described as the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. It consists of a recombinant adeno-associated serotype 9 capsid containing a full-length, wild-type version of the human LAMP2B transgene which, when inserted into the heart cells harboring mutations in the LAMP2 gene, has the potential to fully restore cardiac function at its root, Rocket 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