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Story summary
- U.S. Otsuka Pharmaceutical Development & Commercialization, Inc. launches an early access program for ulefnersen.
- The program supplies ulefnersen to genetically confirmed FUS-ALS patients unable to join trials.
- Ulefnersen is a drug delivered intrathecally that binds FUS pre-mRNA to reduce mutant protein.
- The FDA granted Fast Track and Orphan status, with EMA and Swissmedic also granting Orphan designation.
- Physicians must apply for early access because Otsuka cannot accept direct patient requests.