FDA issues RTF to Harmony Biosciences for IH drug

PLYMOUTH MEETING, Pa. - Harmony Biosciences Holdings , Inc. (NASDAQ: NASDAQ:HRMY), a biopharmaceutical company with strong financials including a 78.65% gross margin and current revenue of $681.88 million, has received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding their submission for pitolisant, aimed at treating excessive daytime sleepiness in adult patients with idiopathic hypersomnia (IH). Despite this setback, Harmony (JO:HARJ) maintains its 2025 revenue guidance of $820-$860 million. According to InvestingPro analysis, the company’s stock is currently trading near its 52-week high of $41.61.

Jeffrey M. Dayno, M.D., President and CEO of Harmony Biosciences, expressed disappointment but reaffirmed the company’s commitment to advancing pitolisant, particularly the higher-dose Pitolisant HD formulation. The company, which maintains a strong balance sheet with more cash than debt and a healthy current ratio of 3.24, plans to initiate a Phase 3 registrational trial for Pitolisant HD in Q4 2025, targeting a Prescription Drug User Fee Act (PDUFA) date in 2028.

Kumar Budur, M.D., M.S., Chief Medical (TASE:PMCN) and Scientific Officer at Harmony, noted that the upcoming Phase 3 trial design, informed by FDA input, aims to substantiate pitolisant’s benefits for IH patients. The decision follows observations from the Phase 3 INTUNE Study, where patients showed significant improvements on the Epworth Sleepiness Scale, with many achieving sustained normal wakefulness levels for over a year.

Despite the RTF, Harmony continues to progress its diverse pipeline, focusing on sleep/wake and rare neurological disorders. Pitolisant is currently approved in the U.S. as WAKIX® for treating excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and for pediatric narcolepsy patients aged six and older. It is not approved for IH treatment. InvestingPro research reveals 10+ additional key insights about Harmony’s financial health and market position, available in the comprehensive Pro Research Report.

WAKIX® is a selective histamine 3 receptor antagonist/inverse agonist, whose mechanism is not entirely understood but is thought to involve increasing histamine synthesis and release. The drug has received both orphan drug designation and breakthrough therapy designation for narcolepsy treatment aspects.

Harmony Biosciences, founded in 2017 and based in Plymouth Meeting, Pennsylvania, is committed to developing therapies for rare neurological diseases with unmet medical needs. With an excellent InvestingPro Financial Health Score of 3.81 and strong analyst support, the company demonstrates solid fundamentals despite market challenges. The information in this article is based on a press release statement from Harmony Biosciences.

In other recent news, Harmony Biosciences faced a significant regulatory challenge as the FDA issued a Refusal to File letter for its pitolisant submission, intended for treating idiopathic hypersomnia. Despite this setback, Harmony maintains its revenue guidance for 2025, projecting between $820 million and $860 million. Analysts at Deutsche Bank (ETR:DBKGn) have initiated coverage with a Buy rating and a $55 price target, highlighting the company’s successful track record with its narcolepsy treatment, Wakix, and its potential to exceed $1 billion in peak sales. Meanwhile, H.C. Wainwright has reaffirmed its Buy rating and a $75 price target, expressing optimism about Wakix’s future and upcoming milestones, including a possible label expansion in 2025.

Oppenheimer also raised its price target for Harmony Biosciences to $61, maintaining an Outperform rating after the company reported strong preliminary fourth-quarter 2024 results. Harmony’s sales reached $201 million for the quarter, surpassing both Oppenheimer’s and Wall Street’s estimates. The company’s guidance for 2025 revenue, set between $820 million and $860 million, exceeded previous estimates from Oppenheimer and Wall Street. Harmony’s pipeline, including advancements in idiopathic hypersomnia treatments and new formulations of pitolisant, continues to present growth opportunities. The company’s management remains confident in its long-term growth potential, aiming to surpass $3 billion in net revenue.

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