The Food and Drug Administration has approved Takeda’s oveporexton, to be marketed as Orzeyful, for the treatment of narcolepsy type 1, according to an Aug. 6 announcement. The drug is expected to become available later this year following scheduling by the U.S. Drug Enforcement Administration.
Orzeyful is the first orexin receptor 2 agonist approved for market use and is also the first medication indicated to address all manifestations of narcolepsy type 1 rather than individual symptoms, according to the FDA’s announcement. Truist Securities said in a note to investors that "this is the first mechanistically differentiated entrant into a sleep base already contending with once-nightly Lumryz and Wakix, and the FDA’s framing of NT1 as ‘a complete disorder’ rather than a symptom cluster is the more consequential piece." Truist analysts also said that Orzeyful's approval currently applies only to adults.
Jefferies told investors that Orzeyful’s approval suggests, “restoration of orexin signaling is likely to reshape the treatment paradigm,” citing efficacy results from Phase 3 FirstLight and RadiantLight trials showing significant improvements over placebo in cataplexy and quality of life measures. Jefferies analysts said Takeda’s oveporexton improved wakefulness, attention, and other key endpoints “with a high degree of statistical significance.” Wakefulness scores measured by Epworth Sleepiness Scale improved by up to 9.8 points versus placebo; Jefferies said these figures bring patients “close to the normal range.”
Jefferies added that Orzeyful compares “favorably with currently available narcolepsy therapies.” Harmony Biosciences’ daily pill Wakix—another FDA-approved drug—showed Epworth Sleepiness Scale score improvements up to 3.1 points relative to placebo; Harmony reported $261.3 million in second quarter revenue for Wakix, marking a 30% year-on-year increase.
Jefferies noted Orzeyful's efficacy edge as its key differentiator but also highlighted its "manageable abuse potential," suggesting it may receive a Schedule IV designation from DEA—a less restrictive category than some other narcolepsy drugs—which "could modestly support physician adoption and market access."
Takeda considers Orzeyful one of three major product launches during what CEO Julie Kim called a “year of transition.” The company also plans launches for rusfertide (for polycythemia vera) under FDA review with an expected decision later this year, and psoriasis pill zasocitinib with an application planned within Takeda's current fiscal year.
