Drugs

Capricor’s Duchenne Cell Therapy BLA Resubmitted

FDA has accepted for review a Capricor Therapeutics BLA resubmission for its experimental cell therapy deramiocel, intended to treat heart complications in patients with Duchenne muscular dystrophy. FDA classified the filing as a Class 2 resubmission and set a new user fee review action target date of 8/22.

The BLA resubmission follows Capricor’s submission of additional clinical data from its HOPE-3 trial after FDA issued a complete response letter on the original BLA 7/2025. The company says the agency has not identified any new potential review issues since accepting the updated filing.

Capricor CEO Linda Marbán said in a statement that results from the HOPE-3 study and other clinical evidence support the therapy’s potential as a first-in-class treatment for Duchenne muscular dystrophy affecting both skeletal and cardiac muscle.

Deramiocel is described as an investigational, first-in-class allogeneic cell therapy designed to preserve cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy, a rare and fatal genetic disease marked by progressive muscle degeneration. The therapy consists of cardiosphere-derived cells that act primarily through exosomes, which are believed to modulate immune responses and reduce fibrosis, 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