Biologics

BrainStorm Cell Therapy Rejected by Panel

FDA’s Cellular, Tissue, and Gene Therapy Advisory Committee 9/27 voted 17 to 1 against approving a BrainStorm Cell Therapeutics BLA for NurOwn, an investigational mesenchymal stem cell therapy for treating amyotrophic lateral sclerosis (ALS, and also known as Lou Gehrig’s Disease).

Panel members mostly agreed with FDA concerns expressed in a briefing document that the clinical data do not demonstrate efficacy and survival was worse at study completion for subjects who received the therapy (see earlier story). Johns Hopkins University professor Caleb Alexander said the single study submitted “didn’t achieve evidence of efficacy for either the primary or secondary endpoints.” He noted that FDA has offered guidance where products can be approved based on data from a single study that was supported by other confirmatory evidence, but not when the trial misses in its demonstration of efficacy.

Disagreeing with vote, Brainstorm said: “the totality of data presented for NurOwn today provide a compelling case for approval, with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response.” It said NurOwn’s poor showing in the study was likely due to a “floor effect, which confounds measurement of disease progression in patients with more advanced disease. A thorough analysis of NurOwn Phase 3 data shows evidence of clinically meaningful effectiveness in ALS participants who have not progressed to advanced levels of disease progression.”

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