Biologics

Under Protest, Brainstorm Resubmits BLA for NurOwn

BrainStorm Cell Therapeutics says it has refiled a BLA under the agency’s “file-over-protest procedure” for NurOwn, a stem cell therapy for treating amyotrophic lateral sclerosis (ALS and also known as Lou Gehrig’s Disease). On 11/10/2022, the agency issued a refusal to file letter for BrainStorm’s BLA. At the time, the company did not disclose the reason FDA gave. However, in 2021 FDA released an unprecedented notice (see story) saying that despite high hopes in the ALS community for NurOwn, Phase 3 trial data did not support its proposed clinical benefit. None of the trial’s primary or secondary endpoints were met, the notice said, with no statistically significant difference between the active and placebo groups.

A file-over-protest submission is not commonly used, and a recent JAMA article noted that it has been used just 10 times over the past decade and no approvals were granted for those submissions. Brainstorm says FDA has acknowledged the submission and confirmed that an advisory committee meeting will be convened to discuss the amended submission. “The FDA provided us with more than one path to an [advisory committee] for NurOwn,” it says. “Our goal has always been to make NurOwn available to people living with ALS as quickly as possible, therefore we chose the file-over-protest pathway since this offered the fastest path…”

BrainStorm says the NurOwn technology platform (autologous MSC-NTF cells) “represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders.” MSC-NTF cells are described as “autologous, differentiated bone-marrow-derived mesenchymal stem cells (MSC) that secrete neurotrophic factors (NTFs), regulatory miRNAs that demonstrate neuroprotection and immunomodulation in preclinical models.”

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