On Wednesday, Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for pitolisant for excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH).

Harmony’s 2025 net revenue guidance of $820 million-$860 million remains unchanged.

“We are disappointed with this short-term setback, but our long-term strategy for pitolisant in IH remains firmly on track with our focus on Pitolisant HD,” said Jeffrey M. Dayno, President and Chief Executive Officer of Harmony Biosciences. “We are on track to initiate a Phase 3 registrational trial of Pitolisant HD in IH in Q4 2025, with a target PDUFA date in 2028.”

Also Read: Neurology-Focused Harmony Biosciences Gains Confidence With Wakix Sales Surge, Strategic Pipeline Expansion, Bullish Analyst Says

Although the primary outcome for excessive daytime sleepiness (EDS) between pitolisant and placebo did not reach statistical significance in the randomized withdrawal phase of the Phase 3 INTUNE Study, the data from the open-label phase showed that patients experienced improvements on the Epworth Sleepiness Scale that were five times greater than what is recognized as clinically meaningful.

Furthermore, most patients in the long-term extension study achieved normal levels of wakefulness and sustained this response beyond one year.

Pitolisant is marketed as Wakix in the U.S. for EDS or cataplexy in adult patients with narcolepsy and for EDS in pediatric patients 6 years of age and older with narcolepsy. It is not currently approved for IH.

In January, the company reported preliminary net product revenue for the fourth quarter of approximately $201 million, compared to $168.4 million a year ago and the consensus of $198.6 million.

The average number of patients on Wakix increased by approximately 300 sequentially to approximately 7,100 for the quarter ended December 31, 2024.

Needham analyst Ami Fadia maintains Harmony Biosciences with a Buy and lowers the price target from $52 to $50.

Price Action: HRMY stock is down 6.50% at $36.55 at last check Wednesday.

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This article Harmony Biosciences Stock Falls On FDA Setback For Pitolisant In Sleeping Disorder originally appeared on Benzinga.com

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