Biologics

FDA Panel Votes Down Capricor's Duchenne Cell Therapy

An FDA advisory committee voted 9 to 3 against recommending approval for Capricor Therapeutics' investigational cell therapy deramiocel, for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD), dealing a significant setback to the company's approval bid ahead of an 8/22 user fee review decision target date.

FDA’s Cellular, Tissue and Gene Therapies Advisory Committee considered whether data from Capricor's Phase 3 HOPE-3 trial, together with earlier studies, demonstrated efficacy for the proposed indication. FDA reviewers argued in briefing documents released before the meeting that submitted data failed to meet the pre-specified primary endpoint measuring upper limb function and its key secondary endpoint evaluating changes in left ventricular ejection fraction, with no statistically significant differences observed between the therapy and placebo after 12 months.

Capricor strongly defended its application during the meeting, maintaining that deramiocel has demonstrated clinically meaningful and statistically significant benefits across multiple studies, including improvements in both skeletal muscle and cardiac function. The company said its BLA is supported by positive results from the Phase 3 HOPE-3 trial as well as long-term follow-up from the Phase 2 HOPE-2 program.

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.

In the briefing document, agency reviewers also raised concerns that multiple changes were made to the study's statistical analysis plan after completion of the blinded portion of the trial, including modifications to endpoint definitions, analytical methods and data handling. FDA said those changes were not prospectively agreed upon and considers the resulting analyses to be post hoc.

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