New York-based biotech BrainStorm Cell Therapeutics announced this week that the FDA has set an advisory committee meeting date to evaluate the Biologic License Application (BLA) for its ALS gene therapy treatment, NurOwn.
According to the recent statement, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (ADCOM) will be meeting on September 27, 2023 to review NurOwn’s data, and the agency has set a PDUFA action date of December 8, 2023.
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, and the agency refused to file NurOwn’s BLA.
Earlier this year, the company 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.
In its recent statement, the BrainStorm shared that a detailed analysis of the data revealed promising effectiveness in ALS patients who were not in advanced stages of the disease.
Despite its bumpy road to potential approval, NurOwn revenue could reach an annual total of $273 million by 2037 globally, according to data from GlobalData's Expiry Model.