BrainStorm has also filed an amendment to its Biologics License Application (BLA) for NurOwn for the treatment of amyotrophic lateral sclerosis (ALS). It said that additional information addresses most of the outstanding questions the US Food and Drug Administration (FDA) had posed.
In an unusual step, the company requested that the Center for Biologics Evaluation and Research (CBER) utilize the FDA's file over protest procedure to proceed to an advisory committee meeting (ADCOM) as “expeditiously as possible.”
"The FDA provided us with more than one path to an ADCOM for NurOwn… We chose the file over protest pathway since this offered the fastest path to an ADCOM and regulatory decision relative to other pathways provided by the FDA," said Chaim Lebovits, CEO of BrainStorm.
NurOwn failed its Phase III clinical efficacy endpoint in November 2020. However, BrainStorm maintains the treatment has shown benefit in patients with less advanced disease at baseline.
The company received a refusal to file letter from the US regulator in November last year regarding its BLA for the ALS therapy.
Filing over protest is a procedural move permitted by FDA regulations that allows a company to have its application filed and reviewed when there is a disagreement with regulators over the acceptability of the sponsor's submission.
The date of the advisory meeting has not yet been announced.
Open forum
Stacy Lindborg, BrainStorm's co-chief executive officer, said that securing an ADCOM represents a key step towards the company’s goal of making NurOwn available to individuals living with ALS, who are in urgent need of new, effective therapies.
“The meeting will provide an open forum for BrainStorm and the FDA, together with medical experts, statisticians, and the ALS community, to thoughtfully review all available evidence supporting NurOwn," he added.
Brian Wallach, patient advocate, also welcomed the meeting: “As was said in the petition submitted to the FDA and signed by over 30,000 people living with ALS, caregivers, and advocates, 'an advisory committee meeting will allow for an independent review of NurOwn as well as the data on efficacy and safety. It is critical for all ALS treatments that they be given the chance to proceed to an ADCOM.'
“It is time to let the science have a full, fair, and transparent hearing.”
