OSAKA, Japan & CAMBRIDGE, Mass. — Takeda (TSE:4502/NYSE:TAK) announced that the U.S. Food and Drug Administration (FDA) approved ORZEYFUL™ (oveporexton), an oral orexin receptor 2 (OX2R) agonist, for the treatment of narcolepsy type 1 (NT1, narcolepsy with cataplexy) in adults.* The persistent 24-hour nature of NT1 is driven by orexin deficiency and can...

BOSTON, Mass. — Galibra Neuroscience announced a significant regulatory milestone with the receipt of both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for the company’s investigational gene therapy for succinic semialdehyde dehydrogenase (SSADH) deficiency. SSADH deficiency is a rare inherited disorder...

SEATTLE, Washington — Shape Therapeutics, a leader in RNA editing-based gene therapy, today announced a partnership with the Rett Syndrome Research Trust (RSRT) to advance SHP-401, an investigational one-time gene therapy for Rett syndrome. Under the agreement, RSRT will fund translational studies to evaluate Shape’s RNAfix® guide RNA, designed to leverage...