Despite the regulatory setback, analysts appear optimistic, noting that Harmony’s long-term prospects remain bright given the development of its high-dose formulation of its sleep-regulating drug Wakix.
Harmony Biosciences has been looking to tack a new indication, idiopathic hypersomnia, onto the label of its excessive daytime sleepiness drug Wakix—but the FDA declined to even review the biotech’s application.
On Wednesday, Harmony announced that it had received a Refusal to File (RTF) letter, which the FDA issues to drug sponsors in case of incomplete submissions, informing them that their applications cannot be reviewed. Harmony CEO Jeffrey Dayno in a statement called the regulator’s decision a “short-term setback” for the company but ultimately one that won’t affect the business in the long run.
“Our long-term strategy for [Wakix] in [idiopathic hypersomnia (IH)] remains firmly on track with our focus on” the high-dose (HD) formulation of the drug, Dayno added. “Our long-term business strategy has always been to extend our leadership in sleep/wake through the development of [Wakix] HD . . . which represents the more significant opportunity in IH given its optimized profile.”
Analysts at H.C. Wainwright agreed with Dayno, writing in an investor note late Wednesday that the biotech’s “long-term strategy remains intact,” with the HD formulation of Wakix making up the bulk of the company’s valuation for the research firm. “Thus, while the RTF is a setback,” the analysts wrote, “we continue to view the long-term opportunity favorably.”
The regulatory setback on Wednesday will not affect Harmony’s 2025 financial outlook; the biotech expects $820 million to $860 million in net revenue this year.
Harmony is gearing up to start a Phase III narcolepsy trial for HD Wakix in the fourth quarter of 2025, designed to assess narcolepsy-related fatigue, which the biotech considers a novel endpoint.
Wakix is an antagonist of the histamine-3 receptor, which is a known player in promoting wakefulness, though the drug’s exact mechanism of action has not yet been completely elucidated, as per its label. The FDA first cleared Wakix in 2019 for adults with narcolepsy suffering from excessive daytime sleepiness.
In October 2023, Harmony released topline data from the Phase III INTUNE study, demonstrating that Wakix elicited treatment responses in 83% of patients with idiopathic hypersomnia, a rare neurological disorder characterized by excessive daytime sleepiness, sleep inertia and brain fog. The drug, however, missed its primary endpoint of a significant four-week improvement in daytime sleepiness, as measured by the Epworth Sleepiness Scale.
