Biologics

Capricor Gets FDA OK on Duchenne BLA Refiling

Capricor Therapeutics says it has reached agreement with FDA on the design and role of its HOPE-3 trial as it works to resubmit its BLA for Deramiocel and its use in treating Duchenne muscular dystrophy (DMD). The update follows a Type A meeting with the agency, convened after it received an FDA complete response letter in July. According to Capricor, regulators agreed that HOPE-3 — a pivotal Phase 3 study now completed — can serve as the “additional study” requested in the CRL. The company expects to report topline data from the trial in mid-fourth quarter and include the results in its resubmission.

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 says.

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.

The original BLA, which was granted a priority review in March, is based on data from the HOPE-2 trial, an open-label extension study, and natural history comparisons using FDA-funded datasets.

DMD is a progressive genetic disease that leads to severe muscle degeneration and heart failure. There is currently no cure, and treatment options remain limited. Cardiomyopathy is a leading cause of death in affected patients, according to the company.

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