FDA approves first-in-class drug to treat narcolepsy Two new FDA approvals, a CRISPR program discontinued, discussions of a major merger, and more led the news this week. Written byDDN editorial team

FDA approves first-in-class drug to treat narcolepsy Two new FDA approvals, a CRISPR program discontinued, discussions of a major merger, and more led the news this week. Written byDDN editorial team https://www.drugdiscoverynews.com/fda-approves-first-in-class-drug-to-treat-narcolepsy-17416 Takeda Pharmaceutical received FDA approval for its narcolepsy type 1 drug, Orzeyful, on Wednesday. The oral drug targets the root cause of narcolepsy — a lack of the neuropeptide orexin in the brain — by activating orexin receptor 2. The approval was based on two large Phase 3 trials showing that the drug led to statistically significant improvements in daytime sleepiness, cataplexy, and health-related quality of life. “The FDA approval of ORZEYFUL marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment,” said Julie Kim, President and CEO of Takeda in the press release. The approval follows years of developing narcolepsy drugs at Takeda, with the company stopping a clinical program back in 2021 due to liver toxicity issues. – Allison Whitten