BrainStorm Cell Therapeutics, a New York-based biotech, announced this week that after years of back and forth with the FDA, the agency has agreed to hold an advisory committee meeting to discuss the company’s application for its amyotrophic lateral sclerosis (ALS) treatment, NurOwn.
Two years ago, the FDA’s initial review concluded that data from BrainStorm Cell Therapeutics' phase 3 trial did not sufficiently provide the threshold of substantial evidence to support a marketing application. At the time, the agency said that this recommendation did not have to stop BrainStorm from proceeding with a BLA submission.
But last November, the FDA refused to file NurOwn’s Biologics License Application after it failed to meet pre-specified primary outcomes in its phase 3 trial. Now, the company has submitted an amendment to the BLA responding to the agency’s concerns and requested that the FDA's biologics unit utilizes a regulatory process called File Over Protest to advance its request to an AdComm meeting as soon as possible. File Over Protest is a request by an applicant to the agency to file their application even though a refuse to file action on their application has already been taken by the FDA.
NurOwn works by extracting mesenchymal stem cells from the patient's bone marrow and then cultivating them in a laboratory to enhance their ability to secrete neurotrophic factors, the signaling molecules that stimulate nerve cell growth.
Despite its arduous journey to potential approval, NurOwn revenue could reach an annual total of $273 million by 2037 globally, according to data from GlobalData's Expiry Model.
