FDA Schedules Second Advisory Committee Meeting for AMX0035 in ALS

The committee will meet on Sept. 7, 2022, to discuss additional analyses of data from the Amylyx’s clinical studies. The updated PDUFA goal date is Sept. 29, 2022.

The FDA has scheduled a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee on Sept. 7, 2022, to review the new drug application (NDA) for Amylyx Pharmaceutical’s AMX0035 (sodium phenylbutyrate and taurursodiol). This is the second meeting of the committee to discuss this application and the data relating to AMX0035 to treat patients with amyotrophic lateral sclerosis (ALS).

This meeting will discuss the additional analyses of data from the company’s clinical studies. As a result, FDA extended the Prescription Drug User Fee Act (PDUFA) target action date for the AMX0035 NDA to Sept. 29, 2022, from the original date of June 29, 2022. At the first advisory committee meeting in March 2022 in which six of the 10 members voted no on the question of whether the open-label extension of the phase 2 CENTAUR trial established a conclusion that AMX0035 is effective in the treatment of patients with ALS.

Related: FDA Extends Review of ALS Therapy

“We remain confident in the data, including the recently published analyses supporting the previously reported functional and overall survival benefit for AMX0035,” Jamie Timmons, M.D., head of scientific communications of Amylyx, said in a press release. There is an urgent unmet need for new and effective treatments in ALS, and our team is working nonstop to continue our mission to end the suffering caused by neurodegenerative diseases.”

The company in May 2022 announced publication of long-term survival analysis of the CENTAUR trial. This analysis used a different model, the rank-preserving structural failure time model, a method frequently used in oncology to account for placebo crossover. Using this model, AMX0035 is estimated to provide a 10.6-month longer median survival duration for participants.

AMX0035 is an oral combination of combination of sodium phenylbutyrate and taurursodiol, which may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum (ER) stress and mitochondrial dysfunction. The therapy is approved in Canada with the brand name Albrioza, and a marketing application is also pending in Europe.