Biopharmaceutical company Amylyx Pharmaceuticals Inc (NASDAQ:AMLX) announced on Friday that it has secured Orphan Drug Designation from the European Commission for AMX0035, a treatment for Wolfram syndrome.

This follows a similar designation from the US Food and Drug Administration (FDA) in 2020.

Wolfram syndrome is rare, progressive, monogenic disease characterised by childhood-onset diabetes, optic nerve atrophy and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus, optic nerve atrophy, diabetes insipidus, deafness, neurogenic bladder and progressive neurologic difficulties. There are currently no drugs approved for Wolfram syndrome, and many people with the disease die prematurely with severe neurological disabilities.

AMX0035 has shown promise in improving pancreatic function and glycaemic control in an interim analysis of the Phase 2 HELIOS study. The drug was generally well-tolerated. Amylyx expects to release topline data from all 12 study participants later this year.