Otsuka Initiates Early Access Program for Ulefnersen, an Investigational FUS-ALS Treatment
Otsuka Pharmaceutical Development & Commercialization, Inc. has launched a global early access program (EAP) for ulefnersen, an investigational RNA-targeted medicine. This program is designed for eligible individuals living with FUS-ALS, a rare and rapidly progressive genetic subtype of amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene. FUS-ALS often has an earlier onset and leads to severe muscle function erosion, impacting independence and ultimately causing premature death. Currently, there are no approved therapies specifically targeting the underlying genetic cause of FUS-ALS. The EAP aims to provide a potential pathway for patients who have a confirmed genetic diagnosis and meet specific eligibility criteria to access ulefnersen outside of clinical trials, where permitted by local regulations. Ulefnersen is designed to reduce the production of toxic FUS protein in motor neurons, which contributes to neurodegeneration in FUS-ALS.
