Amylyx’s ALS Drug Nabs FDA Nod After Rare Regulatory Reversal

After a quest for regulatory approval filled with highs and lows, the FDA approved Amylyx’s amyotrophic lateral sclerosis (ALS) drug, Relyvrio, marking it the third FDA-approved ALS drug in the US. The approval came after an FDA committee voted six to four against the drug’s New Drug Application (NDA) and effectiveness against ALS in March before reconvening in September, flipping the script, voting seven to two that the data established that the drug is effective against ALS. Though the drug is not designed to cure ALS, Amylyx says it can slow progression, paving the way for further ALS research for even more effective therapies in the future. 

Providing ALS Patients Another Option for a Debilitating Disease

ALS is a rare neurodegenerative disease that kills nerve cells that control voluntary movement. As the disease progresses, patients lose the ability to walk, talk, chew, and breathe, ultimately leading to death. More than 90% of ALS patients have no family history of the disease, and the survival time from diagnosis is typically between two and five years. 

With such a dire need for effective therapies to treat ALS, Amylyx designed Relyvrio (formerly AMX0035) as an oral medication consisting of sodium phenylbutyrate and taurursodiol. The two compounds work together to slow nerve cells’ premature death. 

With the FDA’s approval, Amylyx said it plans to price the drug below its competitor, Radicava, another oral ALS medication developed by Japan’s Mitsubishi Tanabe Pharma. Amylyx’s Global Head of Commercial and Chief Commercial Officer, Margaret Olinger, said physicians could begin prescribing Relyvrio immediately before the company will ship the prescriptions out in four to six weeks. 

Merit Cudkowicz, M.D., co-principal investigator on the Phase 2 trial supporting the approval, said, “The approval of a new treatment that helps slow the progression of ALS, preserve physical function, and potentially extend survival has the potential to greatly impact the hundreds of people living with ALS who I currently treat.”

Related Article: Canada Approves Amylyx’s Experimental ALS Drug

A Rocky Path to the Finish Line

The FDA based Relyvrio’s approval on results from the Phase 2 clinical trial called the CENTAUR trial. Despite its current approval status, the FDA did not always find the data convincing. 

Amylyx submitted data from the CENTAUR trial to the regulatory agency earlier this year, but after a committee reviewed the data, the members voted six to four against the data proving the drug’s effectiveness. In light of that news, Amylyx doubled down on the drug’s potential and asked the committee to reconvene after the company gathered more data for the agency to review. 

The committee reconvened earlier this month to comb through all the data again, plus some additional information Amylyx provided. In a rare turn of events, the committee reversed its decision, voting seven to two in support of the data proving the drug’s effectiveness against ALS.  

Based on the data, the FDA determined that Relyvrio demonstrated benefits to patients in the clinical trial. Most noteworthy were the extended survival rates. The regulatory agency recognized that the drug does not cure ALS but can provide patients with more symptom-reduced survival, a valuable commodity when diagnosed with the ravaging disease. 

Canada conditionally approved Relyvrio under the name Albrioza in June this year, provided the company provides more data from a Phase 3 clinical trial at a later date. The European Medicines Agency is still reviewing the drug.

Developing ALS drugs has proven difficult for researchers up to now. Relyvrio is only the third FDA-approved drug on the market alongside Radicava and Sanofi’s Rilutek. Recently, Biohaven Pharmaceuticals announced that its ALS therapy, verdiperstat, did not perform statistically better than the placebo in its Phase ⅔ clinical trial.

Relyvrio’s approval is a huge stepping stone for Amylyx, which has worked tirelessly for years to get to the market to benefit ALS patients with limited treatment options. With the approval, the company hopes its research and development efforts are a jumping-off point for researchers worldwide to develop even more effective ALS therapies. 

Author

Reed Slater