Biologics

Capricor Plans Deramiocel BLA Amendment After FDA Input

Capricor Therapeutics says it plans to amend its BLA for deramiocel to modify its proposed indication after discussions with FDA produced a potential path toward approval of the Duchenne muscular dystrophy therapy based on its Phase 3 trial’s primary endpoint. FDA has indicated it is willing to review an amended application containing 24-month open-label extension data from the HOPE-3 study and additional analyses, with the filing focused on an indication for upper-limb skeletal muscle function, according to the company. FDA would extend the user fee review action date after receiving the amendment, Capricor noted, addng that the timing of the submission has not yet been finalized.

The indication shift follows a 7/29 FDA advisory committee meeting in which members voted 9-3 against a finding that available evidence provided substantial evidence of effectiveness of deramiocel for treating cardiomyopathy in patients with DMD. The committee was asked specifically about the cardiomyopathy indication and was not asked to vote on the HOPE-3 primary endpoint or the overall benefit-risk profile of the therapy.

Capricor CEO Linda Marbán said 8/13 that the company believes the distinction is important because HOPE-3 was designed and powered around a skeletal-muscle endpoint, with upper-limb function as its primary efficacy measure. During the advisory committee meeting, discussion of the upper-limb data was more supportive, according to the company.

The HOPE-3 results showed a statistically significant slowing of upper-limb disease progression measured by the Performance of the Upper Limb 2.0, with a mean treatment difference of 4.55% and a P value of 0.029, the company says.. The full Phase 3 dataset was published in The Lancet in July.

Capricor also disclosed that FDA conducted a Bioresearch Monitoring inspection in July and issued a Form 483 containing one observation. The company has submitted its response and is awaiting FDA feedback.

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