Following approval in Canada in June 2022, the US Food and Drug Administration (FDA) announced overnight that it has approved the therapy RELYVRIO™ (sodium phenylbutyrate and taurursodiol), developed by Amylyx, for the treatment of adults with amyotrophic lateral sclerosis (ALS). RELYVRIO (previously known as AMX0035) significantly slowed the loss of physical function in people living with ALS in a randomised, placebo controlled clinical trial with data from the open-label extension program further supporting clinical benefits of the drug.

This is great news for the MND community as it is the first new treatment since Edaravone was approved in 2017 (this treatment has not been approved for use in Australia) and riluzole in 1995.

The next steps will be pricing for the treatment and approvals in other countries, including Australia. Current pricing in Canada is in excess of $200,000 a year so discussions with authorities over subsiding costs will be critical.

MND Australia have previous made contact with Amylyx around encouraging the company to apply for TGA approval for the treatment to become in Australia. We will be renewing these discussions following the FDA’s positive decision.

More information on this recent development is available via this Amylyx press release and this statement from the FDA.