EMA Recommends Orphan Drug Designation for Tauroursodeoxycholic Acid/Sodium Phenylbutyrate for ALS

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has recommended that tauroursodeoxycholic acid (Turso) /sodium phenylbutyrate (PB) combination (AMX0035; Amylyx Pharmaceuticals, Cambridge, MA) be designated as an orphan medicinal product for the treatment of amyotrophic lateral sclerosis (ALS). Designed to reduce neuronal death and dysfunction, Turso-PB is an investigational, first-in-class, disease-modifying therapy.

“ALS patients worldwide are in need of new therapies,” said Joshua Cohen, cochief executive officer, chairman, and cofounder of Amylyx, “We’re excited about today’s news and look forward to working with the European Medicines Agency on the next steps for AMX0035.”

In late 2019, Amylyx announced that the Centaur clinical trial (NCT03127514) of AMX0035 in individuals with ALS met its primary endpoint, demonstrating that individuals who received AMX0035 showed a statistically significant longer retention of function compared with individuals who received placebo. Function was measured with the ALS functional rating scale-revised (ALSFRS-R). Amylyx is working towards publishing these results in a medical journal as quickly as possible.

The Centaur clinical trial was a pivotal 6 month parallel-group double-blind clinical trial that enrolled 137 participants who were randomly assigned to receive placebo or AMX0035. Upon completion of the 24-week treatment, participants were eligible to enroll in an ongoing open label extension study (NCT03488524) in which all were given access to AMX0035.

AMX0035 is a first in class investigational therapy designed to reduce neuronal death and dysfunction. AMX0035 targets mitochondrial and endoplasmic reticulum dependent neuronal degeneration pathways.