BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced that the U.S. Food and Drug Administration (FDA) has granted a Type A meeting to discuss the contents of a refusal to file letter previously issued regarding the company’s New Biologics License Application (BLA) for NurOwn® for the treatment of ALS. The Type A Meeting has been scheduled to occur on January 11, 2023.
“We look forward to the Type A meeting which will provide an opportunity to discuss the path forward for NurOwn in ALS, including a possible Advisory Committee Meeting,” said Chaim Lebovits, Chief Executive Officer of BrainStorm. “We believe that an Advisory Committee Meeting would serve as an important part of the review process and would provide an open forum for BrainStorm, together with medical experts, statisticians, patients and other members of the ALS community to discuss the body of clinical evidence supporting NurOwn. We intend to provide a further update once we have received the minutes from the meeting, or when we have more clarity on the next steps in the approval process.”
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BrainStorm previously completed a Phase 3 trial in approximately 200 participants with ALS (Cudkowicz et al., 2022 Muscle and Nerve). In an attempt to examine a real-world population, the study enrolled people with more advanced disease than other late-stage ALS trials. In fact, more than a third of these participants with advanced disease entered the trial with one or more dimensions of physical function (e.g., dressing/hygiene, cutting food, walking) starting at the lowest possible score of 0 on the ALSFRS-R; thereby preventing the measurement of further deterioration. A pre-specified subgroup of participants, with baseline ALSFRS-R ≥35, which controls for this ‘scale effect’ showed a trend to a meaningful increase in the clinical response with NurOwn compared to placebo. The secondary endpoint, average ALSFRS-R change from baseline to 28 weeks in this subgroup, was statistically significant (p=0.050, Muscle and Nerve Supplemental File and Muscle and Nerve Erratum).
SOURCE: PR Newswire