STAT+: BrainStorm’s case for ALS approval relies on ‘grossly deficient’ data, FDA finds
The FDA said in newly released briefing documents that it has deep reservations about NurOwn, an investigational ALS treatment from BrainStorm Cell Therapeutics.
The Food and Drug Administration said Monday that it has deep reservations about NurOwn, an investigational ALS treatment from BrainStorm Cell Therapeutics, citing a lack of evidence that the medicine works and the company’s failure to demonstrate that it can properly manufacture the product.
In documents disclosed ahead of a Wednesday public hearing on NurOwn’s application for approval, the FDA detailed its problems with a clinical trial that missed its primary objective and noted that BrainStorm submitted “grossly deficient” information on how the medicine is made, voicing “major concerns that the available data do not meet the statutory standard of substantial evidence of effectiveness to support” approval.
BrainStorm filed for approval over the FDA’s protest, insisting that NurOwn has demonstrated benefits for a subset of ALS patients with milder disease. That conclusion, based on an after-the-fact analysis of the company’s failed pivotal trial, has proved polarizing among patients and physicians, with some arguing that the FDA should exercise flexibility in light of the irrevocably fatal nature of ALS and others demanding BrainStorm conduct another clinical trial to demonstrate its benefits.
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