FDA advisers voted against an experimental ALS treatment pushed by patients. A panel of health experts reviewed a stem cell-based therapy for Lou Gehrig’s disease, the fatal muscle-wasting disease.
The FDA initially refused to review the therapy, but the drug-maker filed its application anyway earlier this year. In their review, FDA called the company’s application “incomplete” and “grossly deficient.”
FDA leaders will make a final decision on whether to approve the treatment later this year.
BrainStorm Cell Therapeutics Inc. released a statement, saying:
“The Committee’s vote was a sad outcome for the ALS community, who have too few options to help manage this merciless and deadly disease,” said Stacy Lindborg, PhD, co-CEO of BrainStorm. “We firmly believe that the totality of data presented for NurOwn today provide a compelling case for approval, with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response. We truly did our best to make the NurOwn data clear to the FDA Advisory Committee. Unfortunately, had more time and opportunity been allowed, many remaining questions posed by Advisory Committee members could have been sufficiently addressed.”
The Associated Press contributed to this report.