BrainStorm Hoping For Advisory Committee After FDA Refuses To File BLA For Its ALS Cell Therapy
BrainStorm, which has a history of antagonizing FDA with direct appeals to patients for NurOwn, tells investors that public elements of an adcomm could be ‘persuasive,’ but first step will be formal meeting request to try to overcome the RTF. Firm sees Relyvrio as template for success.
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A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.
Pink Sheet Drug Review Profile looks at how the agency determined that Amylyx’s ‘conceptual basis’ for use of the drug’s two active ingredients to treat ALS was enough to satisfy the combination drug rule; the sponsor also was allowed to submit certain animal studies and drug-drug interaction data during the course of the NDA review.
Even in a rare disease with no approved treatments, the agency shows once again that without clear efficacy data, FDA has few options for approval.