Biologics

Capricor Reports Data to Support Rejected Deramioce BLA

Capricor Therapeutics says Deramiocel, a cell therapy candidate targeting cardiomyopathy associated with Duchenne muscular dystrophy (DMD), achieved statistically significant improvements in both skeletal muscle function and heart performance in the pivotal Phase 3 HOPE-3 trial. Data from the trial will support a response to a complete response letter (CRL) received from FDA in July, according to the company. The letter cited a lack of “substantial evidence of effectiveness” and called for additional clinical data, according to the company, adding that it also flagged unresolved issues in the BLA’s Chemistry, Manufacturing, and Controls section (see story).

The company says the 106-patient randomized, double-blind, placebo-controlled study met its primary endpoint, showing a 54% slowing of decline in upper-limb function on the Performance of the Upper Limb scale. It also met the trial’s key cardiac endpoint, demonstrating a 91% slowing in deterioration of left ventricular ejection fraction. All type-1–error–controlled secondary endpoints were reported as statistically significant.

In late September, Capricor said it had reached agreement with FDA on the design and role of its HOPE-3 trial as it worked to resubmit its rejected BLA. According to Capricor, regulators agreed that HOPE-3 — could serve as the “additional study” requested in the CRL. The company emphasized that prior to the CRL, most aspects of its BLA — including clinical data, manufacturing, and pre-licensing inspections — had undergone review without major issues. All chemistry, manufacturing and controls questions flagged in the letter have since been addressed, Capricor said at the time.

Deramiocel comprises allogeneic cardiosphere-derived cells, a type of stromal cell that has shown promising immunomodulatory, antifibrotic, and regenerative effects in preclinical and clinical studies, according to the company. These cells work by secreting extracellular vesicles known as exosomes, which can reprogram macrophages to adopt a healing rather than a pro-inflammatory response.

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