FDA Issues Refusal to File for Harmony Biosciences’ Pitolisant for Idiopathic Hypersomnia
On Wednesday, Harmony Biosciences Holdings, Inc. (HRMY) received a setback in its quest to bring pitolisant, an investigational therapy for excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH), to the market. The U.S. Food and Drug Administration (FDA) issued a Refusal to File (RTF) letter for the New Drug Application (NDA) for pitolisant.
What is Idiopathic Hypersomnia?
Idiopathic Hypersomnia (IH) is a chronic, debilitating neurological disorder characterized by excessive daytime sleepiness (EDS), despite adequate nighttime sleep. Patients with IH often experience prolonged sleep periods, multiple naps, and a decreased ability to stay awake during the day. The condition can significantly impact a person’s quality of life, making it difficult to maintain employment, social relationships, and daily activities.
What is Pitolisant?
Pitolisant is an investigational therapy that works by selectively inhibiting the histamine H3 receptor. Histamine plays a role in regulating wakefulness and sleep. By blocking the H3 receptor, pitolisant is thought to increase the amount of histamine available to promote wakefulness. The therapy has already been approved in Europe for the treatment of narcolepsy with cataplexy and EDS in adult patients with IH.
Why did the FDA Issue a Refusal to File?
The FDA’s Refusal to File letter indicates that the agency believes the application is not ready for review. The letter does not provide specific details on the reasons for the refusal. Harmony Biosciences has not yet disclosed the contents of the letter, but it is expected to contain requests for additional information and data from the FDA.
Impact on Harmony Biosciences
The FDA’s Refusal to File letter is a significant setback for Harmony Biosciences. The company had been anticipating a potential approval of pitolisant for the treatment of EDS in adult patients with IH in the first half of 2023. The delay could result in increased development costs, potential loss of revenue, and damage to the company’s reputation.
Impact on Patients
The delay in the approval process for pitolisant is disappointing news for patients with IH. The condition can be debilitating, and current treatment options are limited. Patients continue to seek effective treatments to manage their symptoms and improve their quality of life. The delay in the approval process means that they will have to wait longer for a potential new therapy.
Impact on the Industry
The FDA’s Refusal to File letter for pitolisant highlights the challenges of bringing new therapies to market, particularly in the biopharmaceutical industry. The approval process can be lengthy and costly, and setbacks can occur at any stage. The delay in the approval of pitolisant for IH may discourage investors and potentially slow the pace of innovation in the industry.
Conclusion
The FDA’s Refusal to File letter for Harmony Biosciences’ pitolisant for the treatment of excessive daytime sleepiness in adult patients with idiopathic hypersomnia is a significant setback for the company and the patients who are eagerly awaiting an effective treatment for this debilitating condition. The reasons for the refusal remain unclear, but the company is expected to provide additional information to the FDA to address the concerns raised in the letter. The delay in the approval process may result in increased development costs, potential loss of revenue, and damage to the company’s reputation. Patients with IH will have to wait longer for a potential new therapy, and the delay may also discourage investors and potentially slow the pace of innovation in the industry.
- Harmony Biosciences received a Refusal to File (RTF) letter from the FDA for pitolisant for the treatment of excessive daytime sleepiness in adult patients with idiopathic hypersomnia.
- Idiopathic hypersomnia is a chronic, debilitating neurological disorder characterized by excessive daytime sleepiness.
- Pitolisant is an investigational therapy that works by selectively inhibiting the histamine H3 receptor.
- The FDA’s Refusal to File letter indicates that the application is not ready for review.
- The delay in the approval process may result in increased development costs, potential loss of revenue, and damage to the company’s reputation.
- Patients with IH will have to wait longer for a potential new therapy.
- The delay may also discourage investors and potentially slow the pace of innovation in the industry.
