Takeda’s narcolepsy nod opens orexin doors

 

Orzeyful is the first orexin agonist cleared by the FDA, and is the first narcolepsy therapy that addresses the full host of disease symptoms. It is “likely to reshape the treatment paradigm” for the condition, Jefferies told investors.

The FDA has signed off on Takeda’s oveporexton for the treatment of narcolepsy type 1. The drug will carry the brand name Orzeyful and will be available later this year after the scheduling process with the U.S. Drug Enforcement Administration.

Orzeyful is the first orexin receptor 2 agonist to make it to the market, and is also the first drug indicated to address the full range of manifestations of narcolepsy type 1 (NT1) rather than its individual symptoms, according to the FDA’s Wednesday announcement of the approval.

“To us, 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 Securities wrote to investors on Thursday morning. The firm’s analysts noted, however, that Orzeyful is approved only for adults.

Orzeyful’s approval suggests that “restoration of orexin signaling is likely to reshape the treatment paradigm,” Jefferies told investors in a Wednesday note, pointing to the drug’s efficacy numbers. The Phase 3 pivotal FirstLight and RadiantLight trials showed significant improvements over placebo in areas including cataplexy (sudden muscle weakness) and quality of life.

Wakefulness, as measured by the Epworth Sleepiness Scale, was also significantly better in patients treated with Orzeyful, with scores improving by up to 9.8 points versus placebo, according to the drug’s label. These figures bring patients “close to the normal range,” according to Jefferies.

Orzeyful’s performance, the firm continued, compares “favorably with currently available narcolepsy therapies.”

While Jefferies didn’t name a specific Orzeyful competitor, another FDA-approved drug in the space is Harmony Biosciences’ daily pill Wakix. That drug’s label lists Epworth Sleepiness Scale score improvements of up to 3.1 points relative to placebo. Wakix brought in $261.3 million in the second quarter, a 30% year-on-year increase, Harmony announced Tuesday.

Aside from its efficacy edge, which Jefferies said is the key differentiator of Orzeyful, Takeda’s drug also has a “manageable abuse potential,” the firm noted, suggesting that it is likely to get a Schedule IV designation, one of the DEA’s most lenient classifications.

“If confirmed, this would represent a less restrictive scheduling category” than other narcolepsy drugs, Jefferies added, and “could modestly support physician adoption and market access.”

Orzeyful’s approval is a victory for Takeda, which has tapped the drug as one of three crucial product launches as it enters what new CEO Julie Kim called a “year of transition.” The two other assets are Protaganist-partnered polycythemia vera candidate rusfertide, which is under FDA review with a target decision date in the third quarter of the calendar year, plus the psoriasis pill zasocitinib, for which an application is slated to occur in Takeda’s current fiscal year.

https://www.biospace.com/fda/takedas-narcolepsy-nod-opens-orexin-doors