Harmony Biosciences Faces Regulatory Challenge With Pitolisant
Harmony Biosciences Experiences Regulatory Setback
Recently, Harmony Biosciences Holdings, Inc. (NASDAQ: HRMY) faced a significant hurdle when the U.S. Food and Drug Administration (FDA) issued a Refusal to File (RTF) letter for pitolisant. This was intended for the treatment of excessive daytime sleepiness (EDS) in adults diagnosed with idiopathic hypersomnia (IH).
Company's Revenue Guidance Remains Steady
Despite this short-term setback, Harmony's financial outlook remains robust, with a net revenue guidance for 2025 set between $820 million and $860 million. This consistency in forecast reflects the company's enduring strategy and market position.
CEO Comments on Future Plans
Jeffrey M. Dayno, President and CEO of Harmony Biosciences, expressed disappointment regarding the FDA decision. However, he reassured stakeholders that the initiative surrounding pitolisant for IH is still on course. "We are gearing up to start a Phase 3 registrational trial for Pitolisant HD in IH by the fourth quarter of 2025, targeting a potential approval by 2028," he stated.
Clinical Study Insights
The Phase 3 INTUNE Study's initial results showed that while EDS outcomes comparing pitolisant to placebo did not achieve statistical significance, the open-label phase highlighted notable improvements in patient evaluations on the Epworth Sleepiness Scale. These findings suggested responses that were significantly above what clinical standards acknowledge as meaningful.
Long-term Benefits Evident
Additional data from long-term studies demonstrated that a majority of patients who participated in the trial were able to sustain normal wakefulness over extended periods, showcasing the potential effectiveness of pitolisant in treating this condition.
Wakix – A Cornerstone Product
Pitolisant, known commercially as Wakix in the U.S., is already approved for managing EDS or cataplexy in adult narcolepsy patients, as well as for EDS in pediatric narcolepsy patients aged six and above. Its existing market presence bolsters Harmony's credibility, even as the company seeks expanded indications for IH.
Revenue Performance Insights
In the latest financial report, Harmony recorded preliminary net product revenue of around $201 million for the last quarter, up from approximately $168.4 million during the same period the previous year. This exceeded market expectations, which were pegged at $198.6 million.
Patient Growth Patterns
The uptake of Wakix continued to rise, with the number of patients using the medication increasing by about 300 sequentially, reaching approximately 7,100 by the end of the prior quarter. This growth signifies a positive trend for the company amidst recent challenges.
Analyst Perspectives on Stock Performance
Needham analyst Ami Fadia has maintained a Buy rating for Harmony Biosciences. However, the analyst has adjusted the price target from $52 to $50 in light of the latest developments.
Current Stock Movement
As a result of these recent events, HRMY stock saw a drop of 6.50%, closing at $36.55 according to the latest updates. Investors are keenly observing the company's next steps and its implications for future earnings.
Frequently Asked Questions
What was the main setback for Harmony Biosciences?
The main setback was an FDA Refusal to File letter for pitolisant intended for treating excessive daytime sleepiness in idiopathic hypersomnia.
Is Harmony's revenue guidance affected by this news?
No, Harmony's revenue guidance for 2025 remains unchanged, set between $820 million and $860 million.
What are the future plans for pitolisant in IH?
The company plans to initiate a Phase 3 registrational trial for Pitolisant HD in IH in Q4 2025, targeting a potential approval around 2028.
How did the recent trial results impact patient perceptions?
Even though the primary outcome lacked statistical significance, many patients showed substantial improvements on the Epworth Sleepiness Scale during the open-label phase.
What is the current stock price for HRMY?
The HRMY stock is currently down 6.50%, valued at $36.55 during the latest check.
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