(Reuters) -The U.S. Food and Drug Administration will reconvene a meeting of its outside experts to review Amylyx Pharmaceuticals’ application for its amyotrophic lateral sclerosis (ALS) treatment, the company said on Tuesday.
A panel of outside advisers to the U.S. FDA in March voted six to four against approving the company’s oral drug, AMX0035, saying Amylyx’s clinical study data failed to establish it was effective against ALS, a neurodegenerative disease.
The FDA has since asked for more information from the company and delayed its deadline for deciding on the drug by three months to Sept. 29.
Shares of the company, which said the next FDA meeting on Sept. 7 will focus on additional analyses from Amylyx’s clinical studies, jumped about 13% to $21.91 in early trading.
ALS, whose cause is largely unknown, leads nerve cells in the brain and spinal cord to break down, affecting physical function, resulting in severe disability and even death.
The company’s submission to the FDA is based on a 24-week placebo-controlled mid-stage study of 137 participants with ALS. Amylyx recently started a larger trial in about 600 patients globally, whose completion is not expected until 2024.
AMX0035 was approved in Canada last month with certain conditions including release of data from the ongoing global late-stage study as well as additional studies.
While most ALS treatments currently focus on supportive care, two FDA-approved drugs riluzole and Mitsubishi Tanabe Pharma’s Radicava modestly slow progression of the disease, according to the Institute for Clinical and Economic Review (ICER).
About 25,000 people in the United States suffer from ALS, as per ICER.
Reporting by Mrinalika Roy in Bengaluru.
Source: Read Full Article