A coalition of ALS patients and families has filed a 309‑page Citizens’ Petition with the FDA calling for accelerated approval of NurOwn, a stem cell therapy developed by BrainStorm Cell Therapeutics. The petition brings together over a decade of clinical and real-world evidence, including data from expanded access and right-to-try programs, according to the company.
Among cited results, the petition claims 100 percent five‑year survival among Expanded Access Program participants—compared to approximately 20 percent in natural history ALS cohorts—and median tracheostomy‑free survival of seven years. The document also outlines statistically significant changes in 23 cerebrospinal fluid biomarkers and reports up to 85 percent slowing in disease progression. Additionally, the petition details respiratory improvements, such as extended delay in non‑invasive ventilation and stabilization of forced vital capacity, the company said.
The petition requests evaluation via three FDA approval pathways: traditional approval based on survival data, accelerated approval using surrogate markers, and conditional approval under frameworks tailored to rare diseases. It further asks the FDA’s Center for Biologics Evaluation and Research to conduct a full review, recommending priority voucher deployment and linking approval to a Phase 4 study and data registry. The petitioners emphasise the urgent need given ALS’s fatal prognosis and highlight recent FDA policy commitments to expedite therapies for rare diseases.
