This section highlights FDA-related milestones and regulatory updates for drugs developed by Harmony Biosciences (HRMY).
Over the past two years, Harmony Biosciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
BP1.15205, orexin-2, pitolisant, WAKIX, and ZYN002. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
BP1.15205 - FDA Regulatory Timeline and Events
BP1.15205 is a drug developed by Harmony Biosciences for the following indication: OX2R agonist drug candidate.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BP1.15205
- Announced Date:
- June 11, 2025
- Indication:
- OX2R agonist drug candidate
Announcement
Harmony Biosciences Holdings, Inc. announced that topline clinical data is anticipated in 2026
AI Summary
Harmony Biosciences Holdings, Inc. recently shared promising preclinical data for BP1.15205, a highly potent and selective OX2R agonist aimed at treating narcolepsy and other central disorders of hypersomnolence. The data shows significant wake-promoting effects and a reduction in cataplexy in a mouse model of narcolepsy type 1. Researchers are optimistic about BP1.15205, noting its high oral bioavailability and potential for once-daily dosing, while maintaining a strong safety profile based on a 3‐month GLP toxicity study. A first-in-human study is planned to begin in the second half of 2025. Importantly, Harmony Biosciences anticipates that topline clinical data will be available in 2026, marking a significant step forward in the development of this innovative therapy for individuals with narcolepsy and related sleep disorders.
Read Announcement- Drug:
- BP1.15205
- Announced Date:
- June 11, 2025
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- 2025-H2
- Indication:
- OX2R agonist drug candidate
Announcement
Harmony Biosciences Holdings, Inc. announced that A first-in-human study is planned to start in 2H 2025.
AI Summary
Harmony Biosciences Holdings, Inc. has announced a significant milestone in its drug development program with plans to start a first-in-human study in the second half of 2025. The study will evaluate BP1.15205, an innovative and potentially best-in-class orexin 2 receptor (OX2R) agonist. Preclinical data has shown promising results, including marked wake-promoting effects and a reduction in cataplexy-like episodes in animal models. These findings support the drug’s potential to treat narcolepsy and other central disorders of hypersomnolence.
This oral drug candidate has demonstrated a favorable safety profile in early toxicology studies, with no concerning adverse events noted at high doses. Topline clinical data from the first-in-human trial is expected in 2026, underscoring the company’s commitment to advancing new therapies for sleep-wake disorders.
Read Announcement- Drug:
- BP1.15205
- Announced Date:
- June 11, 2025
- Indication:
- OX2R agonist drug candidate
Announcement
Harmony Biosciences Holdings, Inc announced the presentation of preclinical pharmacological effect data for BP1.15205, an investigational, highly potent, and potentially best-in-class orexin 2 receptor (OX2R) agonist, which demonstrated significant wake-promoting and cataplexy-suppressing effects in a standard transgenic mouse model of narcolepsy type 1.
AI Summary
Harmony Biosciences Holdings announced new preclinical data for BP1.15205, a highly potent and potentially best-in-class orexin 2 receptor (OX2R) agonist. In a transgenic mouse model of narcolepsy type 1, BP1.15205 showed significant wake-promoting effects and reduced cataplexy episodes at low oral doses. The findings suggest that the drug may offer flexible dosing options to treat narcolepsy and other central disorders of hypersomnolence. The compound was shown to be selective with over 600-fold preference for OX2R compared to OX1R, and it demonstrated strong pharmacological benefits without concerning adverse effects.
This promising preclinical data supports BP1.15205’s potential as a novel treatment option and underpins future development, including a planned first-in-human study set to begin in the second half of 2025, with topline clinical results expected in 2026.
Read Announcement
orexin-2 - FDA Regulatory Timeline and Events
orexin-2 is a drug developed by Harmony Biosciences for the following indication: for inducing sleep and the treatment of insomnia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- orexin-2
- Announced Date:
- December 3, 2024
- Indication:
- for inducing sleep and the treatment of insomnia.
Announcement
NLS Pharmaceutics Ltd is pleased to share preclinical data demonstrating the potential of its dual orexin receptor agonist (DOXA) platform.
AI Summary
NLS Pharmaceutics Ltd announced promising preclinical data for its dual orexin receptor agonist (DOXA) platform. The study focused on two new compounds, AEX-41 and AEX-2, which are designed to target both orexin-1 and orexin-2 receptors while also inhibiting cathepsins. In tests using orexin knockout mice, AEX-41 enhanced wakefulness and reduced REM sleep duration, indicating its potential to treat narcolepsy symptoms. This dual-action approach may offer both immediate symptom relief and the possibility of altering the disease process itself. The findings also suggest that the DOXA platform could be useful for other neurological disorders. NLS plans to extend its research by investigating cathepsin inhibition in animal models of neuroinflammation, paving the way for future clinical trials that could broaden treatment options for sleep and neurodegenerative disorders.
Read Announcement- Drug:
- orexin-2
- Announced Date:
- October 1, 2024
- Indication:
- for inducing sleep and the treatment of insomnia.
Announcement
Harmony Biosciences will be providing a comprehensive pipeline update at its Investor Day event today, showcasing new data from its orexin-2 receptor agonist, next-generation pitolisant programs, and long-term extension data from its pitolisant program in idiopathic hypersomnia.
AI Summary
Harmony Biosciences is set to deliver a comprehensive pipeline update during its Investor Day event. The company will showcase new data on its promising orexin‐2 receptor agonist, which is based on a novel chemical scaffold and has demonstrated higher potency than other similar compounds. Additionally, Harmony will share insights from its next-generation pitolisant programs, highlighting progress that could enhance treatment options for sleep disorders. A key part of the update is the presentation of long-term extension data from its pitolisant program targeting idiopathic hypersomnia, where sustained improvements in patient wakefulness have been observed over more than one year of treatment. This event underscores Harmony Biosciences’ commitment to advancing innovative therapies that address critical unmet needs in neurological conditions, aiming to create value for both patients and shareholders.
Read Announcement
pitolisant - FDA Regulatory Timeline and Events
pitolisant is a drug developed by Harmony Biosciences for the following indication: For the treatment of excessive daytime sleepiness.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- pitolisant
- Announced Date:
- February 19, 2025
- Indication:
- For the treatment of excessive daytime sleepiness
Announcement
Harmony Biosciences Holdings, Inc announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for pitolisant for the treatment of excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH).
AI Summary
Harmony Biosciences announced that it received an FDA Refusal to File (RTF) letter for its supplemental New Drug Application for pitolisant to treat excessive daytime sleepiness in adult patients with idiopathic hypersomnia. This decision reflects the FDA’s concerns about the submission’s content, even though the company remains confident in pitolisant’s overall benefit-risk profile for this patient group.
The company expressed disappointment with this setback but reaffirmed its long-term commitment to advancing innovative therapies. Harmony is focusing on Pitolisant HD, an improved, higher-dose formulation, and plans to initiate a Phase 3 registrational trial in Q4 2025 with a potential FDA decision targeted for 2028. Despite this regulatory hurdle, Harmony remains steadfast in its strategy to meet the unmet needs of patients with sleep disorders.
Read Announcement- Drug:
- pitolisant
- Announced Date:
- August 6, 2024
- Indication:
- For the treatment of excessive daytime sleepiness
Announcement
Harmony Biosciences Holdings, Inc announced that the next generation pitolisant-HD development program is advancing and on track towards an expected PDUFA date in 2028.
AI Summary
Harmony Biosciences is making strong progress with its next generation pitolisant-HD development program. Recent pilot pharmacokinetic data show a promising improvement of about 20% in relative bioavailability and reduced variability compared to the current highest labeled dose of WAKIX®. The company is working to optimize the formulation further by testing doses up to twice the current maximum to create an improved product profile. This enhanced profile, which features a gastro-resistant coating and targets additional symptoms such as fatigue, may offer better overall benefits for patients with narcolepsy.
The advancement of this pitolisant-HD program remains on track, with a PDUFA decision expected in 2028. Harmony Biosciences believes the program will extend the pitolisant franchise well beyond 2040. These efforts support the company’s broader goal of addressing unmet medical needs and expanding treatment options in the sleep disorder market.
Read Announcement- Drug:
- pitolisant
- Announced Date:
- June 5, 2024
- Indication:
- For the treatment of excessive daytime sleepiness
Announcement
Harmony Biosciences presented data from its Phase 2 signal detection study showing that pitolisant reduced excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic dystrophy Type 1 (DM1).
AI Summary
Harmony Biosciences recently shared promising Phase 2 study data showing that pitolisant reduced excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic dystrophy Type 1 (DM1). In an 11‐week study, patients treated with pitolisant experienced greater improvements in EDS and fatigue scores compared to those who received a placebo. Notably, the higher dose of pitolisant produced a stronger signal of effectiveness.
Since over 80% of DM1 patients report challenging EDS and fatigue, these findings offer hope for a new treatment option. Based on this encouraging signal, Harmony Biosciences plans to move forward with a pivotal Phase 3 study using a next-generation formulation of pitolisant. This new formulation is designed to optimize the treatment’s benefits, potentially improving the quality of life for people battling DM1 and related sleep/wake disorders.
Read Announcement
WAKIX (pitolisant) - FDA Regulatory Timeline and Events
WAKIX (pitolisant) is a drug developed by Harmony Biosciences for the following indication: Excessive daytime sleepiness or cataplexy in adults with narcolepsy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- WAKIX (pitolisant)
- Announced Date:
- June 24, 2024
- Indication:
- Excessive daytime sleepiness or cataplexy in adults with narcolepsy
Announcement
Harmony Biosciences announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) tablets for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy.
AI Summary
Harmony Biosciences announced that the FDA has approved its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) tablets to treat excessive daytime sleepiness (EDS) in pediatric narcolepsy patients aged 6 years and older. This approval followed a successful Phase 3 study that evaluated the safety and effectiveness of the drug in children with narcolepsy, with or without cataplexy. The FDA’s decision, granted through a priority review, recognizes the need for a non-scheduled treatment alternative in a population that previously had few options beyond controlled substances.
Now, WAKIX becomes the first FDA-approved non-scheduled treatment option available for pediatric patients suffering from narcolepsy-related EDS, providing a promising new approach to improving wakefulness and quality of life for children with this rare sleep disorder.
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ZYN002 - FDA Regulatory Timeline and Events
ZYN002 is a drug developed by Harmony Biosciences for the following indication: In Fragile X Syndrome.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ZYN002
- Announced Date:
- April 8, 2025
- Indication:
- In Fragile X Syndrome
Announcement
Harmony Biosciences Holdings, Inc announced the presentation of updated data from its Open-Label Extension (OLE) study (ZYN2-CL-017) evaluating the safety and effectiveness of ZYN002 in children, adolescents, and adults with Fragile X syndrome (FXS).
AI Summary
Harmony Biosciences announced updated findings from its Open-Label Extension study (ZYN2-CL-017) evaluating ZYN002 for treating Fragile X syndrome (FXS) in children, adolescents, and adults. The study examined the safety and effectiveness of the investigational treatment, showing that more than 60% of patients experienced meaningful improvements in irritability-related symptoms over consecutive visits. Such improvements highlight the potential of ZYN002 to address key behavioral challenges in FXS, a condition that currently has no FDA-approved treatments.
These encouraging results will be presented at the American Academy of Neurology 2025 Annual Meeting during the “Hot Topics in Child Neurology” session. Harmony Biosciences is optimistic that these findings pave the way for ZYN002 to become the first approved treatment for FXS, supporting the company’s commitment to meeting the significant unmet medical needs of the FXS community.
Read Announcement
