Drugs

BrainStorm Gets FDA Alignment on CMC Issues

BrainStorm Cell Therapeutics says it has reached “alignment” with FDA on chemistry, manufacturing, and controls (CMC) aspects of the company’s upcoming Phase 3b clinical trial for NurOwn, its investigational therapy for treating amyotrophic lateral sclerosis (ALS). A recent Type C meeting between the company and FDA helped the company reach an agreement on the CMC isssues, which was one of the last hurdles before the clinical trial could proceed, BrainStorm says. “As with any cell therapy product, there are additional complexities in the manufacturing process, and it is important that we have alignment in advance with all the relevant groups within the FDA,” it says.

In April, BrainStorm and FDA reached an agreement on a Special Protocol Assessment (SPA) agreement for the trial. The SPA “provided clarity on the design and endpoints of the trial, significantly de-risking the regulatory aspects of the program,” the company says.

BrainStorm submitted the SPA request following an in-person meeting that it held with FDA in December after the company withdrew its BLA for NurOwn, a move the company says was coordinated with the agency. A BrainStorm statement at the time said the agency had invited it to request an expedited face-to-face meeting to discuss a path forward for NurOwn, including an additional pivotal trial.

BrainStorm expects to begin the Phase 3b study later this year after reviewing the protocol with investigators, securing study site IRB approvals, and engaging with appropriate members of the ALS community. The trial (Study BCT-006-US) will be a two-part, multicenter study designed to assess the efficacy and safety of NurOwn in patients who are earlier in the course of their disease and having the onset of ALS symptoms, the company says. The first part of the study is a double blind, placebo-controlled trial for 24 weeks. All eligible patients who complete the first part will have the option of entering the second part, an open-label extension where all participants will receive three repeated intrathecal injections of NurOwn once every eight weeks.

The company says the NurOwn technology platform (autologous MSC-NTF cells) “represents a promising investigational therapeutic approach to targeting disease pathways important in neurological disorders. MSC-NTF cells are harvested from each person with ALS and are manufactured using an innovative and proprietary process to secrete neurotrophic factors to target specific neurodegenerative diseases.”

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