Pharma Focus Europe

Amylyx Pharmaceuticals Announces FDA Approval of RELYVRIO™ for the Treatment of ALS

Friday, September 30, 2022

Amylyx Pharmaceuticals, Inc. has received FDA approval for RELYVRIO™ (sodium phenylbutyrate and taurursodiol) for the treatment of amyotrophic lateral sclerosis (ALS) in adults. The drug demonstrated a significant slowing of physical function loss in ALS patients during a randomized, placebo-controlled clinical trial. RELYVRIO can be used as a monotherapy or in combination with existing approved treatments.

The Co-CEOs of Amylyx, Joshua Cohen and Justin Klee, expressed their excitement about the FDA approval and gratitude towards the ALS community for their support and inspiration. They emphasized the company's commitment to ending the suffering caused by neurodegenerative diseases and acknowledged that there is still more work to be done.

ALS is a fatal neurodegenerative disorder characterized by the death of motor neurons in the brain and spinal cord, leading to muscle function deterioration, loss of movement and speech, respiratory paralysis, and eventual death. The majority of ALS cases are sporadic, with no clear family history, and the disease affects approximately 29,000 people in the U.S.

Leading ALS advocacy organizations, including The ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation, and Team Gleason, expressed their support for the FDA approval of RELYVRIO. They believe that the drug offers the potential for increased functional independence and extended time for people living with ALS, which is especially significant considering the rapid progression of the disease and its limited treatment options.

The approval of RELYVRIO is based on data from the CENTAUR clinical trial, which included 137 participants with ALS. The trial consisted of a 6-month randomized, placebo-controlled phase and a long-term follow-up phase. Detailed data from the trial were published in respected medical journals.

The most common adverse events associated with RELYVRIO were diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions were more frequent during the initial three weeks of treatment but occurred throughout the study.

Prominent researchers and principal investigators involved in the CENTAUR trial, including Sabrina Paganoni, M.D., Ph.D., and Merit Cudkowicz, M.D., expressed their enthusiasm for the approval of a new treatment option that can slow ALS progression, preserve physical function, and potentially extend survival. They highlighted the positive impact it could have on their patients and their families.

Amylyx aims to ensure quick access to RELYVRIO for eligible ALS patients in the U.S. Physicians can prescribe the drug immediately, and specialty pharmacies are expected to begin filling prescriptions and shipping RELYVRIO within the next four to six weeks.

Amylyx made the decision to price RELYVRIO below the latest FDA-approved product for ALS, considering input from various stakeholders within the ALS community in the U.S. To support access, the company has established the Amylyx Care Team (ACT) Support Program. ACT provides a dedicated point of contact for ALS patients prescribed RELYVRIO, offering guidance, assistance with insurance navigation, financial support options, education, and resources. Individuals can reach out to the ACT team via phone or email to receive personalized support.

Overall, the approval of RELYVRIO represents a significant milestone in the battle against ALS, offering new hope and treatment options for patients and their families.

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