FDA to review citizen’s petition for BrainStorm ALS therapy

A coalition of amyotrophic lateral sclerosis patients have filed a 309-page citizens' petition with the FDA requesting approval of NurOwn, an autologous MSC-NTF cell therapy developed by BrainStorm Cell Therapeutics for the treatment of ALS.

In a press release, BrainStorm acknowledged that the petition, which requests a new review of the data supporting NurOwn, will provide a critical opportunity to reaffirm the therapy’s potential.

"We respect the FDA's independent review process and welcome its consideration of this request," said Chaim Lebovits, President and CEO of BrainStorm. "We continue to stand firmly behind the scientific integrity of BrainStorm's data and remain committed to working collaboratively with the FDA and the ALS community to advance the development of safe and effective therapies."

The company's proprietary NurOwn platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection.

BrainStorm recently announced new survival data from 10 participants who had previously completed a phase 3 clinical trial and subsequently entered its Expanded Access Program (EAP). Notably, participants survived more than five years from ALS symptom onset – well above published estimates suggesting that only 10% of people diagnosed with ALS reach that benchmark. The median survival in the group was 6.8 years.

In May, BrainStorm announced that the FDA has cleared the company to initiate its phase 3b clinical trial under FDA Special Protocol Assessment, a formal process in which companies can meet with the FDA to agree on trial design.

But it’s been a long regulatory path for NurOwn. In February 2021, after reviewing data from a phase 3 ALS trial, senior leadership at the FDA advised BrainStorm that its clinical data did not meet the threshold of substantial evidence to support a BLA. In November 2022, the agency proceeded to reject NurOwn's BLA, citing phase 3 trial outcomes. BrainStorm submitted an amendment addressing the agency’s concerns and requested fast-tracking through a process known as ‘file over protest’ to secure an AdComm meeting. Almost a year later, in October 2023, BrainStorm opted to pull its BLA, cutting back resources and laying off 30% of its staff after an FDA AdComm voted 17-1 against approving NurOwn, noting concerns over the company’s data and its inability to demonstrate how they would safely manufacture the treatment.