Harmony Biosciences Receives U.S. Food and Drug Administration Approval for WAKIX® (pitolisant) for the Treatment of Cataplexy in Pediatric Narcolepsy

Key Terms

cataplexy medical

Cataplexy is sudden, brief loss of muscle control triggered by strong emotions such as laughter, surprise or anger; it can range from drooping facial muscles to full body collapse while the person remains conscious. For investors, cataplexy matters because its severity and chronic nature drive demand for treatments, influence clinical trial design and regulatory review, and affect market size, pricing and healthcare costs in neurology and sleep disorder portfolios.

narcolepsy medical

A chronic neurological condition that causes sudden, uncontrollable episodes of deep sleep and extreme daytime drowsiness, like a phone that unexpectedly shuts off during the day. It matters to investors because it defines the market need for treatments, influences clinical trial results and regulatory decisions, and can drive revenue or risk for companies developing medications, devices, or diagnostics tied to sleep disorders.

supplemental new drug application regulatory

A supplemental new drug application is a request submitted to regulatory authorities to make changes to an existing approved medication, such as adding new uses, strengths, or formulations. For investors, it signals that a pharmaceutical company is seeking approval for new product developments or expanded applications, which can impact the company's future sales, market potential, and stock value.

snda regulatory

A SNDA (Subordination, Non‑Disturbance and Attornment Agreement) is a legal pact among a property owner’s lender, the owner’s tenants, and sometimes the landlord that sets who keeps lease rights if the property is sold or a mortgage is enforced. Think of it as a rulebook that decides whether a tenant can stay and keep paying rent or must answer to a new owner after a foreclosure. For investors, an SNDA matters because it protects predictable rental income, clarifies who has priority on claims against a property, and therefore affects a property’s value and the security of related loans.

excessive daytime sleepiness medical

A chronic condition in which a person repeatedly struggles to stay awake or experiences overwhelming sleep episodes during normal daytime activities, not merely the occasional tiredness everyone feels. Investors care because it affects large groups of patients, drives demand for medical tests, treatments and supportive services, and shapes potential market size and healthcare spending—imagine a smartphone that keeps losing charge during the day and needs a new type of battery.

controlled substance regulatory

A controlled substance is a drug or chemical whose manufacture, possession, use and distribution are restricted by government law because of potential for abuse, addiction or harm. For investors it matters because these rules shape a company’s ability to sell products, obtain licenses, win approvals and avoid fines or criminal risk — like a speed limit that constrains how fast a business can grow or exposes it to penalties if violated.

pediatric exclusivity regulatory

Pediatric exclusivity is a regulatory reward that gives a drug maker extra protected selling time—typically a six‑month extension—if the company conducts studies showing the medicine is safe and effective for children. For investors, it matters because that extra time is like a temporary shield that delays generic competition and can materially extend revenue from a product, affecting a drug’s cash flow, valuation and investment returns.

regulatory exclusivity regulatory

A period during which regulators prevent competitors from relying on a drug or device maker’s safety and effectiveness data to win approval for their own rival product, creating a temporary market shield beyond or instead of a patent. For investors, this delay in direct competition can extend a product’s revenue runway and improve predictable cash flow, like giving a seller exclusive access to a popular storefront for a set time before others can move in.

PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)-- Harmony Biosciences (Nasdaq: HRMY) today 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 cataplexy in pediatric patients 6 years of age and older with narcolepsy. WAKIX is now the first-and-only FDA-approved non-scheduled treatment for pediatric and adult narcolepsy patients with or without cataplexy.

“We are excited to achieve this important milestone for pediatric narcolepsy patients who experience cataplexy,” said Kumar Budur, M.D., M.S., Chief Medical and Scientific Officer of Harmony Biosciences. “With this approval, clinicians now have the option to prescribe WAKIX to treat excessive daytime sleepiness, cataplexy, or both, in patients 6 years and older with narcolepsy.”

WAKIX was approved by the FDA in August 2019 for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy, with approval expanded to include cataplexy in adult patients in October 2020. The FDA approved WAKIX for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy in June 2024.

“WAKIX is now the first-and-only FDA-approved treatment option for both pediatric and adult narcolepsy patients with or without cataplexy that is not scheduled as a controlled substance, an important distinction that supports its clinical utility,” said Jeffrey M. Dayno, M.D., President and Chief Executive Officer of Harmony Biosciences. “We continue to advance our efforts toward obtaining pediatric exclusivity for WAKIX, which, if granted, would add an additional six months of regulatory exclusivity for this growing franchise. Our sights are now set on further growing, extending and expanding the value of pitolisant through additional indications with our next-generation formulations, which have utility patents filed out to 2044.”

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 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 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 higher concentrations of pitolisant (e.g., patients with hepatic or renal impairment). Monitor patients with hepatic or renal impairment 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 the treatment of excessive daytime sleepiness or cataplexy in pediatric patients less than 6 years of age 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.

The maximum recommended dosage is lower in patients who are CYP2D6 poor metabolizers because these patients have higher pitolisant concentrations than CYP2D6 normal metabolizers and may have increased risk of adverse events.

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 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 2026 net product revenue for WAKIX, expectations for the growth and value of WAKIX, plans to submit an NDA for pitolisant GR; plans to conduct trials, collect or receive data, or continue investigating any of our product candidates or potential indications; our plans to extend the pitolisant franchise into the 2040s; 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 and, if approved, our other product candidates; 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; our research and development plans, including our efforts to explore the therapeutic potential of pitolisant in additional indications, including pitolisant GR and pitolisant HD; 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 and, if approved, our other product candidates; the timing of, and our ability to obtain, regulatory approvals for pitolisant for other indications, including pitolisant GR and pitolisant HD, 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 the significant costs and required management time as a result of operating as a public company. 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.

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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.