Drugs

FDA Rejects Capricor Duchenne Cell Therapy

FDA has issued Capricor Therapeutics a complete response letter on its BLA for Deramiocel, a cell therapy candidate targeting cardiomyopathy associated with Duchenne muscular dystrophy (DMD). 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. Capricor notes that many of these concerns had already been addressed in earlier communications. However, it says those materials were not reviewed due to the timing of the letter.

Capricor plans to request a Type A meeting with the FDA to clarify next steps and says it intends to submit data from its ongoing Phase 3 HOPE-3 trial later this year to strengthen the evidence base.

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 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

Read more

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