An article in BioSpace announced that the FDA’s CNS committee met twice in recent months to discuss the merits of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). Within weeks of the committee’s endorsing the drug with a seven to two vote, the FDA gave its approval.
AMX0035 is now the third drug to be approved for ALS. It will be marketed under the brand name Relyvrio.
Clinical data evidenced a slowing of the progression of ALS with a median survival of 18.8 months.
About ALS
Amyotrophic lateral sclerosis is known as Lou Gehrig’s disease named after the popular baseball player. ALS kills nerve cells that are responsible for controlling voluntary muscles. Patients no longer have the ability to manage the movement of their muscles causing the muscles to weaken, leading to paralysis. The disease is fatal. Death, usually from respiratory failure, occurs within three to five years from the date initial symptoms are discovered.
Currently, there are between twelve to fifteen thousand people in the U.S. with ALS. About five thousand to six thousand cases are diagnosed each year.
The data used for the Relyvrio approval was based on the CENTAUR (NCT03127514) Phase II trial’s assessment of median survival. A total of 137 patients participated. When the patients’ clinical decline was compared to that of the placebo cohort, there was a significantly slower rate of decline. The drug extended patients’ survival by six months or more.
Relyvrio is the third drug approved by the FDA to treat ALS after approval in 1995 of riluzole and edaravone’s approval in 2017.
About the PHOENIX Trial
A larger study called PHOENIX is expected to announce results in 2025. Amylyx anticipates the PHOENIX Phase III trial will confirm the extent of Relyvrio’s benefit to patients.
Since the protocol leading to a vote usually requires the results of two independent clinical trials, each with hundreds of participants, the addition of the larger Phase III trial raised the advisory board’s confidence level as they voted their approval.
The primary objectives of the PHOENIX trial are efficacy and safety. Another important factor is the potential effect of Relyvrio on the progression of ALS over a thirty-eight-week period.
Amylyx CEOs Cohen and Klee stated that healthcare providers have the authorization to start writing prescriptions for Relyvrio through a support program that Amylyx is in the process of implementing. The company describes it as a dedicated one point of contact. Details will be announced shortly.
AMX0035 (Relyvrio) was initially authorized in Canada under the name Albrioza. The co-CEOs sent a statement to BioSpace saying that their goal is to see that every patient eligible for Relyvrio will be able to gain access to the medication. They emphasized the urgency and stressed that teams are standing by ready to assist.
New York’s BrainStorm, also vying for FDA approval for its drug to treat ALS, announced its intention to submit a Biologics License Application for its product, NurOwn.
At the same time, BrainStorm submitted a corrected analysis that the company believes will add to its previous conclusions.
Both companies are committed to fulfilling their promise to ALS patients.
