ALS Community Rallies Behind BrainStorm's NurOwn, Urging FDA Reconsideration

In a significant development for the amyotrophic lateral sclerosis (ALS) community, patients and advocates have submitted a Citizens' Petition to the U.S. Food and Drug Administration (FDA), requesting the approval of BrainStorm Cell Therapeutics' cell therapy NurOwn. This move comes in the wake of new survival data and represents a renewed push for a treatment that has faced regulatory hurdles in recent years.

Petition Highlights New Evidence

The Citizens' Petition, supported by BrainStorm Cell Therapeutics, points to "new evidence and totality of evidence" as grounds for the FDA to reconsider NurOwn's approval. Central to this appeal is recently reported data from an expanded access program (EAP) involving Phase III trial participants. According to BrainStorm, nine out of ten EAP participants survived more than five years from ALS symptom onset, with a median survival of 6.8 years. This significantly outperforms the typical survival rate for ALS patients, which is estimated at around 10% past the five-year mark.

Patient petitioners emphasized the unprecedented nature of these results, citing "100% five-year survival versus 20% ALS natural history" and extended periods of progression-free survival ranging from a few months to 17 months in patients receiving NurOwn.

Regulatory Challenges and Clinical Trials

NurOwn's journey through the regulatory process has been fraught with setbacks. In 2021, the FDA advised BrainStorm that its Phase III trial results were insufficient to support a Biologics License Application (BLA). The company's subsequent attempts to file a BLA based on reanalyzed data were met with a Refusal to File Letter from the FDA in 2022.

Despite these challenges, BrainStorm has persisted in its efforts to bring NurOwn to market. The company is now preparing to launch a Phase IIIb trial, with 15 clinical sites selected and Clinical Trial Agreements in the process of being executed. The trial's commencement is pending secured funding, according to a BrainStorm representative.

Industry Context and Future Outlook

The push for NurOwn's approval comes at a critical time for the ALS community. The recent withdrawal of Amylyx's Relyvrio from U.S. and Canadian markets following a failed Phase III trial has left a significant gap in treatment options. However, the field remains active, with companies like Korro Bio, Coya Therapeutics, and QurAlis exploring novel targets against ALS.

As the FDA considers the Citizens' Petition and BrainStorm prepares for its next clinical trial, the ALS community watches with keen interest. The outcome of this latest development could have far-reaching implications for ALS treatment and the regulatory pathway for innovative cell therapies.