Harmony Biosciences Receives Refusal to File Letter from FDA for Pitolisant in Idiopathic Hypersomnia
On February 16, 2023, Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) announced that it had received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for pitolisant in the treatment of excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH).
What is Idiopathic Hypersomnia and Pitolisant?
Idiopathic hypersomnia is a rare, chronic neurological disorder characterized by excessive daytime sleepiness and prolonged nighttime sleep. Pitolisant is an orally administered, non-opioid, selective histamine H3 receptor antagonist/inverse agonist. It was designed to improve wakefulness and reduce daytime sleepiness in patients with IH.
The Impact on Harmony Biosciences
Harmony Biosciences stated that they were disappointed with this setback but that their long-term strategy for pitolisant in IH remains unchanged. The company’s 2025 net revenue guidance of $820-$860 million remains unchanged. The RTF letter does not affect the ongoing development programs for pitolisant in narcolepsy and Prader-Willi syndrome. Harmony plans to work closely with the FDA to address the issues raised in the RTF letter and resubmit the application as soon as possible.
The Impact on Patients
For patients with idiopathic hypersomnia, the receipt of an RTF letter for pitolisant is a significant disappointment. Many of these patients have been eagerly awaiting an approved treatment for their condition. The FDA’s concerns regarding the application could delay the availability of pitolisant as a treatment option for this patient population.
The Impact on the World
The delay in the approval of pitolisant for the treatment of idiopathic hypersomnia could have far-reaching implications. Idiopathic hypersomnia is a rare disorder, but it affects an estimated 1 in 2,000 to 1 in 10,000 people worldwide. The absence of approved treatments for this condition can result in significant impairment in daily functioning and reduced quality of life. Furthermore, the delay could set back efforts to increase awareness and understanding of idiopathic hypersomnia and the importance of developing effective treatments for this patient population.
Conclusion
The receipt of an RTF letter from the FDA for pitolisant in the treatment of idiopathic hypersomnia is a setback for Harmony Biosciences and the patients who have been eagerly awaiting an approved treatment for this condition. However, the company remains committed to working with the FDA to address the concerns raised in the RTF letter and resubmit the application as soon as possible. The delay could impact the lives of those with idiopathic hypersomnia, but the long-term goal remains the development of effective treatments for this rare and often misunderstood disorder.
- Harmony Biosciences received an RTF letter from the FDA regarding pitolisant for IH
- Pitolisant is an orally administered, non-opioid, selective histamine H3 receptor antagonist/inverse agonist
- Impact on patients: delay in treatment availability
- Impact on the world: setback for awareness and understanding of idiopathic hypersomnia
- Harmony remains committed to working with the FDA to address concerns and resubmit the application
