Amylyx Pharmaceuticals, a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035 for the treatment of Wolfram syndrome.

“There is a critical unmet need for people living with Wolfram syndrome, a rare genetic disease in which many patients die prematurely with severe neurological disabilities,” said Wolfram syndrome expert Fumihiko Urano, M.D., Ph.D., a Professor of Medicine and of Pathology and Immunology at Washington University School of Medicine in St. Louis.

“Endoplasmic reticulum (ER) dysfunction is a critical component of Wolfram syndrome. AMX0035 is designed to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome.”

The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S. Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval.

“We are grateful to the advocacy groups, Dr. Urano and his team, and the dedicated parents who are working with us to determine if AMX0035 can help patients and families affected by Wolfram syndrome,” said Kent Leslie, Global Head of R&D and Chief Scientific Officer at Amylyx.

“Orphan drug designation will support us as we explore AMX0035 in treating this rare and underserved population living with a life-threatening disease that has no approved therapies. This designation is an important milestone as we continue to develop AMX0035 for the potential treatment of neurodegenerative diseases. We look forward to continuing our evaluation of AMX0035 in animal models of Wolfram syndrome and, hopefully soon, in patients.”

AMX0035 is currently being investigated in people living with ALS and Alzheimer’s disease.

Wolfram syndrome is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties.

Genetic and experimental evidence suggest that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of Wolfram syndrome. The prognosis of Wolfram syndrome is poor, and many patients die prematurely with severe neurological disabilities.