AMX0035 receives orphan drug designation for Wolfram syndrome
Click Here to Manage Email Alerts
The FDA granted orphan drug designation to AMX0035, an investigational neuroprotective therapy, for Wolfram syndrome, a rare neurodegenerative disease that causes severe neurological disabilities and results in premature death.
“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,” Fumihiko Urano, MD, PhD, professor of medicine and pathology and immunology at Washington University School of Medicine in St. Louis, said in a press release. “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.”
Characteristic symptoms of Wolfram syndrome, an autosomal recessive disease, include childhood-onset diabetes, optic nerve atrophy and neurodegeneration. The disease is associated with a poor prognosis; many patients die prematurely with severe neurological disabilities.
Current research demonstrates that ER dysfunction is a critical pathogenic component of Wolfram syndrome. AMX0035, which is also being investigated in ALS, is intended to decrease neuronal death and dysfunction; it targets ER and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.