Regulators Still To Get To Grips With Neurodegenerative Outcome Measures

Outcomes measures in the neurodegenerative disease space are not yet well understood by regulators, according to Amylyx, which recently received a negative opinion from the EMA for its amyotrophic lateral sclerosis drug, Albrioza.

Path in forest — Regulators approach new outcome measures in neurodegenerative diseases • Source: Shutterstock

Following a negative opinion from the EMA’s human medicines committee, the CHMP, for its orphan drug Albrioza (sodium phenylbutyrate/ursodoxicoltaurine) for amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals, Inc. told the Pink Sheet that regulators do not yet fully understand the best outcomes measures for neurodegenerative diseases.

Justin Klee and Joshua Cohen, co-CEOs of Amylyx, said that while experts were aligned on the best way to measure...