RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Japan)

Structure/Sequence

Molecular FormulaC17H27Cl2NO

InChIKeyXLFKECRRMPOAQS-UHFFFAOYSA-N

CAS Registry903576-44-3

Clinical Trials associated with Pitolisant Hydrochloride

NCT07219485

/ Enrolling by invitationPhase 3

An Open-Label Study to Evaluate Safety of Pitolisant in Participants With Prader-Willi Syndrome

The purpose of this study is to assess safety of pitolisant in eligible participants with Prader-Willi syndrome (PWS) who completed the End of Treatment (EOT) visit in a parent study (HBS-101-CL-002 [Open Label Extension (OLE)], HBS- 101-CL-004, or HBS-101-CL-312 OLE).

Start Date20 Aug 2025

Sponsor / Collaborator

Zynerba Pharmaceuticals, Inc.

NCT07051252

/ RecruitingPhase 1

A Phase 1b, Open-Label Study of HBS-201 (Pitolisant Delayed-release) in Adult Participants With Narcolepsy

The purpose of this study is to assess the tolerability of HBS-201 when starting at a therapeutic dose in adult participants with narcolepsy.

Start Date21 May 2025

Sponsor / Collaborator

Zynerba Pharmaceuticals, Inc.

NCT06366464

/ RecruitingPhase 3

A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome

This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome.
Secondary objectives include assessing the impact of pitolisant on:
Irritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech

Start Date28 May 2024

Sponsor / Collaborator

Zynerba Pharmaceuticals, Inc.

100 Clinical Results associated with Pitolisant Hydrochloride

100 Translational Medicine associated with Pitolisant Hydrochloride

100 Patents (Medical) associated with Pitolisant Hydrochloride

246

Literatures (Medical) associated with Pitolisant Hydrochloride

01 Nov 2025·CURRENT OPINION IN PULMONARY MEDICINE

Understanding idiopathic hypersomnia: diagnosis, pathophysiology, and management

Review

Author: Shahzadi, Madeeha ; Thorpy, Michael J ; Monderer, Renee

Purpose of review:

Idiopathic hypersomnia is a chronic and often disabling sleep disorder characterized by excessive daytime sleepiness despite adequate or prolonged nighttime sleep. With recent advances in diagnosis and treatment, this review is timely in addressing evolving approaches to understanding and managing idiopathic hypersomnia, a condition that remains underrecognized and frequently misdiagnosed.

Recent findings:

Idiopathic hypersomnia is clinically distinct from narcolepsy, lacking REM-related features such as cataplexy and hypnagogic hallucinations. Until recently, treatment options for idiopathic hypersomnia were limited and often off-label. The 2021 FDA approval of low-sodium oxybate (LXB) marked the first medication specifically indicated for idiopathic hypersomnia. Additional agents such as modafinil, pitolisant, and traditional stimulants are used off-label with varying efficacy. Ongoing research is exploring promising treatments, including orexin-2 receptor agonists (e.g. ALKS 2680, ORX-750), serdexmethylphenidate, and flumazenil, which offer new hope for personalized management.

Summary:

Growing insights into the pathophysiology and clinical features of idiopathic hypersomnia have led to improved diagnostic clarity and therapeutic innovation. These developments carry significant implications for clinical practice, offering hope for better symptom control and quality of life in affected individuals. Ongoing research is crucial for refining treatment strategies and deepening our understanding of this complex condition.

01 Oct 2025·ARCHIVOS DE BRONCONEUMOLOGIA

Residual Excessive Daytime Sleepiness in Obstructive Sleep Apnoea and New Pharmacological Possibilities for Treatment

Review

Author: Bekki, Amina ; Gómez-Olivas, Jose Daniel ; Oscullo, Grace ; Martínez-García, Miguel Ángel

Excessive daytime sleepiness is one of the most important symptoms in obstructive sleep apnoea and it is often used to decide whether the latter should be diagnosed as a syndrome or considered a priority in diagnosis and treatment, and whether the treatment is effective. Beyond this, one concept of enormous clinical significance has emerged: "residual excessive daytime sleepiness", defined as excessive daytime sleepiness in the context of a diagnosed obstructive sleep apnea that is treatable with continuous positive airway pressure (CPAP) or alternatives to it; it appears as a consequence of poor adaptation to, or refusal of treatment, or even sometimes in situations of good tolerance. Given the direct relationship between excessive daytime sleepiness (usually defined by an Epworth Sleepiness Scale [ESS] value of more than 10 points) and cardiovascular, neurocognitive and quality-of-life disorders, attempts have been made for years to alleviate it with drugs (modafinil, armodafinil, sodium oxybate or amphetamines), to little effect and with a large number of adverse effects. However, unlike their predecessors, two products that have recently appeared on the market - solriamfetol and pitolisant - have achieved clinically significant reductions without any major adverse effects, in most cases. In fact, a Task Force from the European Respiratory Society has even proposed an algorithm for residual excessive daytime sleepiness and its pharmacological treatment in the context of obstructive sleep apnoea. The present review aims to define the importance of excessive daytime sleepiness, especially residual excessive daytime sleepiness in patients with obstructive sleep apnoea, as well as the old and new pharmacological alternatives that have appeared.

Journal of Clinical Sleep Medicine

Pitolisant may lessen not only sleepiness but improve hyperphagia and behavior problems in Prader-Willi Syndrome

Article

Author: Grigg-Damberger, Madeleine

News (Medical) associated with Pitolisant Hydrochloride

03 Dec 2025

·www.biospace.com

Harmony’s Dravet Drug Halves Monthly Seizures in Late-Stage Study

iStock, Only Flags The Phase III, open-label extension study suggests EPX-100 has a “positive” risk/benefit pro the treatment of Dravet syndrome, analysts at H.C. Wainwright & Co. said Tuesday. Harmony Biosciences’ EPX-100 reduced monthly seizures by about half in a Phase III study of patients with Dravet syndrome. “These results increase our confidence in the EPX-100 program,” analysts at H.C. Wainwright & Co. said in a note to investors on Tuesday. The analysts called the drug “a cornerstone of Harmony’s strategy” to complement the company’s excessive sleepiness drug Wakix. The readout, H.C. Wainwright added, “represents a de-risking event for a key pipeline asset” for the biotech. Tuesday’s data come from the open-label extension stage of the Phase III ARGUS study, a randomized, double-blinded and placebo-controlled trial that compared EPX-100 against placebo, looking at countable motor seizures per 28 days, a metric called CMS-28. Results showed that in patients who had been taking EPX-100 for at least 6 months in this extension phase, there was an “approximately 50% reduction” in CMS-28 from baseline. All 29 patients who had completed the double-blind portion of the study continued on to the open-label extension phase, which H.C. Wainwright said is indicative of “a positive patient and physician view of the drug’s risk/benefit profile.” More detailed data from the trial is scheduled to be presented on Dec. 8 at the American Epilepsy Society’s annual conference. Aside from EPX-100’s preliminary efficacy, the ARGUS open-label phase also assessed the drug’s safety profile. There were 28 drug-related side effects and 11 serious adverse events, of which one was “possibly” linked to EPX-100, according to the study investigator. The H.C. Wainwright analysts called the drug’s safety profile “favorable” overall. “While from a small, open-label cohort, this clinically meaningful efficacy signal provides strong validation for the program” in Dravet syndrome, H.C. Wainwright added. Harmony is also running the Phase III LIGHTHOUSE study for EPX-100, this time in Lennox-Gastaut syndrome, a rare and severe type of epilepsy in childhood. Both studies are pivotal, the analysts said, and are set to read out next year. The Dravet win on Tuesday breaks somewhat of a losing streak for Harmony, which in September announced that its cannabidiol candidate ZYN002 failed a late-stage study in fragile X syndrome. In the Phase III RECONNECT trial, patients treated with ZYN002 showed no significant improvement in social avoidance. And though the biotech did not provide specific data at the time, it blamed the miss on a “higher than expected placebo response rate.” In February, the FDA refused to accept Harmony’s supplemental application to expand Wakix into idiopathic hypersomnia. The biotech at the time did not reveal the reasoning behind the refusal.

Phase 3Clinical Result

24 Nov 2025

·ir.harmonybiosciences.com

Harmony Biosciences Announces Positive Results From the Pivotal Bioequivalence Study for Pitolisant Gastro-Resistant Formulation

New Drug Application to be submitted in early 2026; target PDUFA in Q1 2027 Utility patent applications filed with potential exclusivity to 2044 PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Nov. 24, 2025-- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced positive results from its pivotal bioequivalence (BE) study evaluating pitolisant gastro-resistant (GR) formulation. With positive findings from both the pivotal BE and dosing optimization studies, Harmony is on track to submit a New Drug Application (NDA) for pitolisant GR in early 2026, targeting a PDUFA date in Q1 2027. With utility patent applications filed for pitolisant GR with potential exclusivity to 2044, Harmony is well positioned to extend the pitolisant franchise into the mid-2040s. “We are very pleased to have demonstrated bioequivalence for pitolisant GR which, along with the positive findings from our dosing optimization study, confirms its potential as a differentiated next-generation treatment option for narcolepsy patients,” said Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences. “The combination of GR coating and ability to initiate treatment at the therapeutic dose without the need for titration, can potentially result in an enhanced overall treatment experience with pitolisant GR.” The pivotal BE study confirmed that 17.8mg of pitolisant GR is bioequivalent to existing 17.8mg WAKIX® tablets. The pitolisant GR vs. WAKIX AUC and Cmax ratios (%) were 108.46 h*ng/mL (90% CI 103.74 – 113.41) and 99.65 ng/mL (90% CI 91.95 – 108.00), respectively. No new safety or tolerability issues were reported. Importantly, the topline data from the dosing optimization study of pitolisant GR showed 100% of patients successfully initiated treatment at the therapeutic dose of 17.8mg, eliminating the need for dose titration. This progress on pitolisant GR underscores the accelerating momentum of the pitolisant franchise, which is rapidly approaching blockbuster status in narcolepsy alone. Harmony Biosciences’ commitment to innovation is further demonstrated by the development of pitolisant HD, a high-dose, enhanced formulation of pitolisant with an optimized pharmacokinetic profile, designed to deliver greater efficacy for excessive daytime sleepiness and other residual symptoms in narcolepsy and idiopathic hypersomnia. With utility patent applications for both pitolisant GR and pitolisant HD filed to potentially extend exclusivity to 2044, Harmony is well positioned for sustained market leadership and long-term value creation in the sleep/wake therapeutic space. About WAKIX® (pitolisant) Tablets WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. Indications and Usage WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy. Important Safety Information Contraindications WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment. Warnings and Precautions WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval. The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is contraindicated in patients with severe hepatic impairment and not recommended in patients with end-stage renal disease (ESRD). Adverse Reactions In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and at least twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in patients treated with placebo included headache, upper respiratory tract infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash. In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and greater than placebo) for WAKIX were headache (19%) and insomnia (7%). The overall adverse reaction profile of WAKIX in the pediatric clinical trial was similar to that seen in the adult clinical trial program. Drug Interactions Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half. Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required. H1 receptor antagonists that cross the blood-brain barrier may reduce the effectiveness of WAKIX. Patients should avoid centrally acting H1 receptor antagonists. WAKIX is a borderline/weak inducer of CYP3A4. WAKIX may reduce the effectiveness of sensitive CYP3A4 substrates, including hormonal contraceptives. Patients using hormonal contraception should be advised to use an alternative non-hormonal contraceptive method during treatment with WAKIX and for at least 21 days after discontinuing treatment. Use in Specific Populations There is a pregnancy exposure registry that monitors pregnancy outcomes in women who are exposed to WAKIX during pregnancy. Patients should be encouraged to enroll in the WAKIX pregnancy registry if they become pregnant. To enroll or obtain information from the registry, patients can call 1-800-833-7460. The safety and effectiveness of WAKIX have not been established for treatment of excessive daytime sleepiness in pediatric patients less than 6 years of age with narcolepsy. The safety and effectiveness of WAKIX have not been established for treatment of cataplexy in pediatric patients with narcolepsy. WAKIX is extensively metabolized by the liver. WAKIX is contraindicated in patients with severe hepatic impairment. Dosage adjustment is required in patients with moderate hepatic impairment. WAKIX is not recommended in patients with end-stage renal disease. Dosage adjustment of WAKIX is recommended in patients with eGFR <60 mL/minute/1.73 m2. Dosage reduction is recommended in patients known to be poor CYP2D6 metabolizers; these patients have higher concentrations of WAKIX than normal CYP2D6 metabolizers. Please see the Full Prescribing Information for WAKIX for more information. To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. About Narcolepsy Narcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 170,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation (hallucinations and sleep paralysis), which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin/orexin, a neuropeptide in the brain that supports sleep-wake state stability. This disease affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed. About Idiopathic Hypersomnia Idiopathic Hypersomnia (IH) is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness (EDS) despite sufficient or even long sleep time. EDS in IH cannot be alleviated by naps, longer sleep or more efficient sleep. People living with IH experience significant EDS along with the symptoms of sleep inertia (prolonged difficulty waking up from sleep) and 'brain fog' (impaired cognition, attention, and alertness). The cause of IH is unknown, but it is likely due to alterations in areas of the brain that stabilize states of sleep and wakefulness. IH is one of the central disorders of hypersomnolence and, like narcolepsy, is a debilitating sleep disorder that can result in significant disruption in daily functioning. About Harmony Biosciences Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Driven by novel science, visionary thinking, and a commitment to those who feel overlooked, Harmony Biosciences is nurturing a future full of therapeutic possibilities that may enable patients with rare neurological diseases to truly thrive. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, Pa., we believe that when empathy and innovation meet, a better future can begin; a vision evident in the therapeutic innovations we advance, the culture we cultivate, and the community programs we foster. For more information, please visit www.harmonybiosciences.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our full year 2025 net product revenue, expectations for the growth and value of WAKIX, plans to submit an sNDA for pitolisant in idiopathic hypersomnia; our future results of operations and financial position, business strategy, products, prospective products, product approvals, the plans and objectives of management for future operations and future results of anticipated products. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our commercialization efforts and strategy for WAKIX; the rate and degree of market acceptance and clinical utility of pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved, including ZYN002 and EPX-100; our research and development plans, including our plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreements with Bioprojet Société Civile de Recherche (“Bioprojet”); the availability of favorable insurance coverage and reimbursement for WAKIX; the timing of, and our ability to obtain, regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and additional financing needs; our ability to identify, acquire and integrate additional products or product candidates with significant commercial potential that are consistent with our commercial objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellectual property position; loss or retirement of key members of management; failure to successfully execute our growth strategy, including any delays in our planned future growth; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; the significant costs and required management time as a result of operating as a public company; the fact that the price of Harmony's common stock may be volatile and fluctuate substantially; statements related to our intended share repurchases and repurchase timeframe; and macroeconomic effects and changes in market conditions, including the impact of tariffs, inflation and the risk of recession. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 25, 2025, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. View source version on businesswire.com: https://www.businesswire.com/news/home/20251124261066/en/ Harmony Biosciences Investor Contact: Matthew Beck 917-415-1750 matthew.beck@astrpartners.com Harmony Biosciences Media Contact: Cate McCanless 202-641-6086 cmccanless@harmonybiosciences.com Source: Harmony Biosciences Holdings, Inc.

Orphan DrugLicense out/inClinical ResultDrug ApprovalBreakthrough Therapy

19 Nov 2025

·ir.harmonybiosciences.com

Harmony Biosciences Announces Initiation of First-In-Human Study With Potential Best-In-Class Orexin 2 Receptor Agonist (BP1.15205) for Central Disorders of Hypersomnolence

BP1.15205 is a differentiated chemical scaffold, demonstrating high potency and selectivity in preclinical studies with a favorable safety profile and the potential for once-daily dosing Topline clinical data expected in 2026 PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Nov. 19, 2025-- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced dosing of the first participant in a Phase 1 clinical trial of BP1.15205, an investigational, potentially best-in-class, orexin 2 receptor (OX2R) agonist being developed for the treatment of narcolepsy, idiopathic hypersomnia and other central disorders of hypersomnolence. The Phase 1 clinical trial will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of BP1.15205 after single and multiple ascending doses in healthy volunteers and sleep-deprived healthy subjects. Topline data are expected in 2026. “We are very excited to advance BP1.15205 into clinical development based on its compelling preclinical risk-benefit profile,” said Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences. “Based on the potency data for BP1.15205, it has the potential to enable very low, once-daily dosing, which may translate into a favorable risk-benefit profile for patients with central disorders of hypersomnolence.” Earlier this year, Harmony Biosciences presented comprehensive preclinical safety and efficacy data for BP1.15205 at SLEEP 2025 and at the World Sleep Congress. Functional receptor studies confirmed that the asset is a highly potent and selective OX2R agonist with no observed off-target effects. The preclinical data revealed no adverse events of interest, supporting a favorable safety and tolerability profile. “The advancement of our orexin program is an important expansion of our sleep-wake franchise, especially in a market where polypharmacy is the norm,” Dr. Budur added. “We believe orexin agonists will complement our pitolisant-based assets, allowing Harmony to offer additional treatment options that address the complex, individualized needs of patients with narcolepsy and other hypersomnolence disorders.” The Phase 1 clinical trial is being conducted by Bioprojet Pharma in the European Union. Bioprojet originally licensed BP1.15205 from Teijin Pharma. Harmony Biosciences has an exclusive licensing agreement with Bioprojet to develop, manufacture and commercialize BP1.15205 in the United States and Latin American territories. About Narcolepsy Narcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 170,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation (hallucinations and sleep paralysis), which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin/orexin, a neuropeptide in the brain that supports sleep-wake state stability. This disease affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed. About Idiopathic Hypersomnia Idiopathic Hypersomnia (IH) is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness (EDS) despite sufficient or even long sleep time. EDS in IH cannot be alleviated by naps, longer sleep or more efficient sleep. People living with IH experience significant EDS along with the symptoms of sleep inertia (prolonged difficulty waking up from sleep) and 'brain fog' (impaired cognition, attention, and alertness). The cause of IH is unknown, but it is likely due to alterations in areas of the brain that stabilize states of sleep and wakefulness. IH is one of the central disorders of hypersomnolence and, like narcolepsy, is a debilitating sleep disorder that can result in significant disruption in daily functioning. About Harmony Biosciences Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Driven by novel science, visionary thinking, and a commitment to those who feel overlooked, Harmony Biosciences is nurturing a future full of therapeutic possibilities that may enable patients with rare neurological diseases to truly thrive. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, Pa., we believe that when empathy and innovation meet, a better future can begin; a vision evident in the therapeutic innovations we advance, the culture we cultivate, and the community programs we foster. For more information, please visit www.harmonybiosciences.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our full year 2025 net product revenue, expectations for the growth and value of WAKIX, plans to submit an sNDA for pitolisant in idiopathic hypersomnia; our future results of operations and financial position, business strategy, products, prospective products, product approvals, the plans and objectives of management for future operations and future results of anticipated products. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our commercialization efforts and strategy for WAKIX; the rate and degree of market acceptance and clinical utility of pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved, including ZYN002 and EPX-100; our research and development plans, including our plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreements with Bioprojet Société Civile de Recherche (“Bioprojet”); the availability of favorable insurance coverage and reimbursement for WAKIX; the timing of, and our ability to obtain, regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and additional financing needs; our ability to identify, acquire and integrate additional products or product candidates with significant commercial potential that are consistent with our commercial objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellectual property position; loss or retirement of key members of management; failure to successfully execute our growth strategy, including any delays in our planned future growth; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; the significant costs and required management time as a result of operating as a public company; the fact that the price of Harmony's common stock may be volatile and fluctuate substantially; statements related to our intended share repurchases and repurchase timeframe; and macroeconomic effects and changes in market conditions, including the impact of tariffs, inflation and the risk of recession. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 25, 2025, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. View source version on businesswire.com: https://www.businesswire.com/news/home/20251119313237/en/ Harmony Biosciences Investor Contact: Matthew Beck 917-415-1750 matthew.beck@astrpartners.com Harmony Biosciences Media Contact: Cate McCanless 202-641-6086 cmccanless@harmonybiosciences.com Source: Harmony Biosciences Holdings, Inc.

Phase 1License out/in

100 Deals associated with Pitolisant Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Pitolisant Hydrochloride	N07XX11	DB11642

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Excessive Daytime Sleepiness	Canada	ENDO VENTURES LTD	31 Dec 2021
Sleep Apnea, Obstructive	European Union	Bioprojet Pharma SARL	01 Sep 2021
Sleep Apnea, Obstructive	Iceland	Bioprojet Pharma SARL	01 Sep 2021
Sleep Apnea, Obstructive	Liechtenstein	Bioprojet Pharma SARL	01 Sep 2021
Sleep Apnea, Obstructive	Norway	Bioprojet Pharma SARL	01 Sep 2021
Cataplexy	United States	Harmony Biosciences Holdings, Inc.	13 Oct 2020
Narcolepsy	European Union	Bioprojet Pharma SARL	31 Mar 2016
Narcolepsy	Iceland	Bioprojet Pharma SARL	31 Mar 2016
Narcolepsy	Liechtenstein	Bioprojet Pharma SARL	31 Mar 2016
Narcolepsy	Norway	Bioprojet Pharma SARL	31 Mar 2016

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Idiopathic Hypersomnia	NDA/BLA	United States	Harmony Biosciences Holdings, Inc.	-
Prader-Willi Syndrome	Phase 3	United States	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Australia	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Belgium	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Canada	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Denmark	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	France	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Germany	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Italy	Zynerba Pharmaceuticals, Inc.	28 May 2024
Prader-Willi Syndrome	Phase 3	Poland	Zynerba Pharmaceuticals, Inc.	28 May 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04257929 (CTgov)	Phase 2	Prader-Willi Syndrome	65	Pitolisant oral tablets (Double-Blind Treatment Phase Lower Dose Pitolisant)	puvyqskxun(zkwpqguxfd) = tlkathjiey zjgpbljgdo (nhvjejltzm, 1.14) View more	-	22 Oct 2025
				Pitolisant oral tablets (Double-Blind Treatment Phase Higher Dose Pitolisant)	puvyqskxun(zkwpqguxfd) = jrszdhnbyl zjgpbljgdo (nhvjejltzm, 1.07) View more
NCT04257929 (Pubmed \| J Clin Sleep Med)	Phase 2	Prader-Willi Syndrome	65	Lower-dose Pitolisant (8.9/13.35/17.8 mg)	nduofsaant(rlrciugmmb) = ibyztgospb wtgubwwrzc (rkunrbsbys )	Positive	03 Jul 2025
				Higher-dose Pitolisant (17.8/26.7/35.6 mg)	nduofsaant(rlrciugmmb) = djfzvrtqqr wtgubwwrzc (rkunrbsbys )
NCT04886518 (CTgov)	Phase 2	Excessive Daytime Sleepiness \| Myotonic Dystrophy	30	Pitolisant Oral Tablet (Higher Dose Pitolisant)	nladvcnkva(bvsjajusre) = gpdaypepgt crzwabfcji (duwahxfapu, 1.98) View more	-	25 Apr 2025
				Pitolisant Oral Tablet (Lower Dose Pitolisant)	nladvcnkva(bvsjajusre) = hkkbvcsohp crzwabfcji (duwahxfapu, 1.91) View more
JPRN-jRCT2031220432 (Company_Website) Manual	Phase 3	Narcolepsy	-	Pitolisant (Japanese patients)	hsiutpzfhi(wavcofhomk) = the primary efficacy endpoint was achieved. ncbexscnfp (wlvbqiwxrb ) Met	Positive	21 Oct 2024
				Placebo (Japanese patients)
NCT02739568 (HAROSA3, Pubmed)	-	Sleep Apnea, Obstructive	361	替洛利生	mpdpqanuvt(jtthlhbchl): difference = -2.6 (95% CI), P-Value = <0.001	Positive	08 Oct 2024
				安慰剂
MDS2024	Not Applicable	Restless Legs Syndrome	19	Pitolisant + Pitolisant RLS medications	jiflxlakmv(iaixnllgec) = rhfgoalhdg hqklzbiafi (sjeopjsqlc, 5.0)	Positive	27 Sep 2024
NCT02611687 (CTgov)	Phase 3	Cataplexy	110	pitolisant (Pitolisant)	rnmptvmaci(lgdecxpnpu) = yhuqbciqdq yxttdlinym (kuukkqrvol, 1.14) View more	-	19 Sep 2024
				Placebo (Placebo)	rnmptvmaci(lgdecxpnpu) = jpeqsphove yxttdlinym (kuukkqrvol, 1.35) View more
NCT01071876 \| NCT01072968 (Pubmed \| Chest)	Phase 3	Sleep Apnea, Obstructive	512	Pitolisant	tjiqrcvolx(qrdxooyiwc) = lfaxkkpiml qlaegnbsyk (kaadumanva ) View more	Positive	01 Mar 2024
NCT04596267 (CTgov)	Phase 1	Alcohol Use Disorder	9	Pitolisant (Pitolisant)	tctxdxunzg(etraiebmeb) = bmupzhypyn aumnioggmv (jhxkawwelq, 25.759) View more	-	04 Dec 2023
				Placebo (Placebo)	tctxdxunzg(etraiebmeb) = muubogtjhj aumnioggmv (jhxkawwelq, 15.369) View more
WSC2023	Not Applicable	Narcolepsy	110	Pitolisant	ymycetfrog(okpzeohkku) = rrjrhdtety wkeqmaofxl (riyyhclkkj )	Positive	23 Oct 2023
				Placebo	ymycetfrog(okpzeohkku) = etalyjihze wkeqmaofxl (riyyhclkkj )

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