PITOLISANT HYDROCHLORIDE - Generic Drug Details

What are the generic sources for pitolisant hydrochloride and what is the scope of freedom to operate?

Pitolisant hydrochloride is the generic ingredient in one branded drug marketed by Harmony and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Pitolisant hydrochloride has sixty-one patent family members in thirty-one countries.

One supplier is listed for this compound.

Summary for PITOLISANT HYDROCHLORIDE

International Patents:	61
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	55
Clinical Trials:	28
Patent Applications:	9
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for PITOLISANT HYDROCHLORIDE
What excipients (inactive ingredients) are in PITOLISANT HYDROCHLORIDE?	PITOLISANT HYDROCHLORIDE excipients list
DailyMed Link:	PITOLISANT HYDROCHLORIDE at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for PITOLISANT HYDROCHLORIDE

Constraining patent/regulatory exclusivity:

TREATMENT OF EXCESSIVE DAYTIME SLEEPINESS (EDS) IN PEDIATRIC PATIENTS 6 YEARS OF AGE AND OLDER WITH NARCOLEPSY

Dosage:

TABLET;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for PITOLISANT HYDROCHLORIDE

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
University of Oxford	N/A
Harmony Biosciences, LLC	Phase 4
William Ondo, MD	Phase 4

See all PITOLISANT HYDROCHLORIDE clinical trials

Pharmacology for PITOLISANT HYDROCHLORIDE

Drug Class	Histamine-3 Receptor Antagonist/Inverse Agonist
Mechanism of Action	Cytochrome P450 3A4 Inducers Histamine H3 Receptor Antagonists Histamine H3 Receptor Inverse Agonists

Anatomical Therapeutic Chemical (ATC) Classes for PITOLISANT HYDROCHLORIDE

N07XX	Other nervous system drugs
N07X	OTHER NERVOUS SYSTEM DRUGS
N07	OTHER NERVOUS SYSTEM DRUGS
N	Nervous system

Paragraph IV (Patent) Challenges for PITOLISANT HYDROCHLORIDE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
WAKIX	Tablets	pitolisant hydrochloride	4.45 mg and 17.8 mg	211150	7	2023-08-14

US Patents and Regulatory Information for PITOLISANT HYDROCHLORIDE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Harmony	WAKIX	pitolisant hydrochloride	TABLET;ORAL	211150-001	Aug 14, 2019		RX	Yes	No
Harmony	WAKIX	pitolisant hydrochloride	TABLET;ORAL	211150-002	Aug 14, 2019		RX	Yes	Yes
Harmony	WAKIX	pitolisant hydrochloride	TABLET;ORAL	211150-001	Aug 14, 2019		RX	Yes	No	8,486,947

Expired US Patents for PITOLISANT HYDROCHLORIDE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Harmony	WAKIX	pitolisant hydrochloride	TABLET;ORAL	211150-001	Aug 14, 2019	7,169,928
Harmony	WAKIX	pitolisant hydrochloride	TABLET;ORAL	211150-002	Aug 14, 2019	7,169,928
Harmony	WAKIX	pitolisant hydrochloride	TABLET;ORAL	211150-001	Aug 14, 2019	7,910,605

International Patents for PITOLISANT HYDROCHLORIDE

Country	Patent Number	Title	Estimated Expiration
Japan	2014062126	TREATMENT OF SYMPTOMS OF PARKINSON'S DISEASE, OBSTRUCTIVE SLEEP APNEA, DEMENTIA WITH LEWY BODIES, VASCULAR DEMENTIA WITH NON-IMIDAZOLE ALKYLAMINE HISTAMINE H3-RECEPTOR LIGANDS
Norway	20075086
China	101155793	Monohydrochloride salt of 1-[3-[3-(4-chlorophenyl)propoxy]propyl]-piperidine

Supplementary Protection Certificates for PITOLISANT HYDROCHLORIDE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1428820	16C0038	France		PRODUCT NAME: PITOLISANT ET SES SELS PHARMACEUTIQUEMENT ACCEPTABLES TELS QUE LE CHLORHYDRATE; REGISTRATION NO/DATE: EU/1/15/1068 20160331
1428820	122016000073	Germany		PRODUCT NAME: PITOLISANT UND DESSEN PHARMAZEUTISCH VERTRAEGLICHE SALZE, WIE DAS HYDROCHLORID; REGISTRATION NO/DATE: EU/1/15/1068 20160331
1428820	CA 2016 00042	Denmark		PRODUCT NAME: PITOLISANT OG FARMACEUTISK ACCEPTABLE SALTE HERAF, SASOM PITOLISANT HYDROCHLORID; REG. NO/DATE: EU/1/15/1068 20160404

PITOLISANT HYDROCHLORIDE Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Pitolisant Hydrochloride

Introduction

Pitolisant hydrochloride, marketed as WAKIX, is a significant player in the treatment of narcolepsy and other sleep disorders. Developed by Harmony Biosciences, this drug has shown remarkable market performance and continues to evolve with new formulations and indications. Here, we delve into the market dynamics and financial trajectory of pitolisant hydrochloride.

Market Opportunity and Patient Base

Pitolisant hydrochloride has tapped into a substantial market opportunity, particularly in the treatment of adult narcolepsy. With approximately 80,000 patients diagnosed with narcolepsy in the U.S., the drug has seen strong commercial sales driven by organic demand[3].

Revenue Performance

Since its launch in November 2019, WAKIX has achieved impressive revenue milestones. In the third quarter of 2024, the drug generated $186.0 million in net revenue, contributing to a cumulative net revenue of over $2 billion in less than five years[2][3].

Quarterly and Annual Growth

The third quarter of 2024 saw a 16% growth in net product revenue compared to the same period in 2023, primarily due to strong commercial sales of WAKIX. This growth is indicative of the drug's broad clinical utility and its acceptance among healthcare providers (HCPs)[3].

Patient Growth and Utilization

The average number of patients on WAKIX increased by approximately 250 sequentially to about 6,800 for the quarter ended September 30, 2024. This steady increase in patient numbers reflects the drug's efficacy and the expanding reach of Harmony Biosciences' commercial efforts[3].

Expanding Indications

Idiopathic Hypersomnia (IH)

Harmony Biosciences is on track to submit a supplemental new drug application (sNDA) for pitolisant in idiopathic hypersomnia (IH) in the fourth quarter of 2024. New data from the Long-Term Extension study demonstrate robust and sustained efficacy of pitolisant in patients with IH, with significant improvements in the Epworth Sleepiness Scale (ESS), Idiopathic Hypersomnia Severity Scale (IHSS), and Sleep Inertia Questionnaire (SIQ)[3].

Next-Generation Formulations

The company is advancing next-generation formulations of pitolisant, including Pitolisant-HD (high dose) and Pitolisant-GR (gastro-resistant). These formulations are designed to provide enhanced pharmacokinetic profiles and higher dosage ranges to drive greater efficacy. The Pitolisant-HD program, for instance, targets a unique indication for fatigue in narcolepsy and is expected to extend the pitolisant franchise into the mid-2040s[3].

Financial Performance and Outlook

Operating Expenses and Income

In the third quarter of 2024, Harmony Biosciences reported operating expenses that included $25.4 million in research and development, $27.6 million in sales and marketing, and $28.6 million in general and administrative costs. Despite these expenses, the company achieved an operating income of $61.7 million[2].

Revenue Guidance

For 2024, Harmony Biosciences has reiterated its net product revenue guidance of $700 - $720 million, reflecting the continued strong performance of WAKIX and the potential from new indications and formulations[3][5].

Pipeline and Future Catalysts

Late-Stage Pipeline

Harmony Biosciences has a catalyst-rich, late-stage pipeline poised to deliver one or more new launches every year over the next five years. This includes the potential best-in-class, novel orexin-2 agonist, with an Investigational New Drug (IND) application on track for mid-2025[3].

Pediatric Exclusivity and Additional Indications

The company is also working towards pediatric exclusivity to extend WAKIX exclusivity to September 2030. Additionally, Harmony Biosciences has initiated a Phase 3 TEMPO study in Prader-Willi Syndrome (PWS) and plans to start a pivotal Phase 3 trial in 22q11.2 deletion syndrome (22q) in 2025[5].

Competitive Landscape and Leadership

Harmony Biosciences has strengthened its leadership position in the sleep/wake disorder market through the licensing of TPM-1116, a highly potent and selective oral orexin-2 receptor agonist. This move, along with the advancement of its pitolisant franchise, solidifies the company's position as a key player in this therapeutic area[5].

Intellectual Property and Patent Strategy

To extend the pitolisant franchise beyond 2040, Harmony Biosciences has filed provisional patents that could grow the franchise by pursuing additional indications. This strategic approach ensures the company maintains a competitive edge and protects its intellectual property[1][3].

Key Statistics and Highlights

• Revenue Milestone: $2 billion in cumulative net revenue in less than five years[2][3].
• Patient Growth: Approximately 6,800 patients on WAKIX as of Q3 2024[3].
• Efficacy Data: Mean improvement in Epworth Sleepiness Scale (ESS) of ~9 points from baseline in patients with idiopathic hypersomnia[3].
• Pipeline Advancements: Next-generation formulations (Pitolisant-HD and Pitolisant-GR) and new indications (IH, PWS, 22q)[3][5].

Conclusion

Pitolisant hydrochloride, under the brand name WAKIX, has established itself as a dominant force in the treatment of narcolepsy and is poised for further growth with expanding indications and next-generation formulations. Harmony Biosciences' strong financial performance, coupled with a robust pipeline and strategic intellectual property management, positions the company for continued success in the sleep/wake disorder market.

Key Takeaways

• Strong Revenue Growth: WAKIX has surpassed $2 billion in cumulative net revenue in less than five years.
• Expanding Indications: New data supports the use of pitolisant in idiopathic hypersomnia, with submissions planned for Q4 2024.
• Next-Generation Formulations: Pitolisant-HD and Pitolisant-GR are advancing to extend the franchise beyond 2040.
• Robust Pipeline: Harmony Biosciences has a catalyst-rich pipeline with multiple new launches expected over the next five years.
• Intellectual Property Strategy: Provisional patents filed to protect and extend the pitolisant franchise.

FAQs

What is the current market performance of WAKIX?

WAKIX has generated $186.0 million in net revenue for the third quarter of 2024, contributing to a cumulative net revenue of over $2 billion since its launch in 2019[2][3].

What are the new indications being explored for pitolisant?

Harmony Biosciences is on track to submit an sNDA for pitolisant in idiopathic hypersomnia (IH) in Q4 2024 and is also exploring indications in Prader-Willi Syndrome (PWS) and 22q11.2 deletion syndrome (22q)[3][5].

How is Harmony Biosciences extending the pitolisant franchise?

The company is advancing next-generation formulations (Pitolisant-HD and Pitolisant-GR) and has filed provisional patents to extend the franchise beyond 2040[1][3].

What is the significance of the orexin-2 agonist in Harmony Biosciences' pipeline?

The potential best-in-class, novel orexin-2 agonist is expected to be a key catalyst, with an IND application on track for mid-2025, further solidifying the company's leadership in sleep/wake disorders[3].

How does Harmony Biosciences' financial performance reflect its market position?

The company's strong financial results, including a 16% growth in net product revenue in Q3 2024 and an operating income of $61.7 million, reflect its robust market position and the success of WAKIX[2][3].

Sources

1. Harmony Biosciences. Harmony Biosciences Acknowledges US Food and Drug Administration (FDA) [Press Release]. Retrieved from https://ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-acknowledges-us-food-drug-administration-fda
2. Harmony Biosciences. Harmony Biosciences Reports Strong Third Quarter 2024 Financial Results and Highlights Catalyst-Rich, Late-Stage Pipeline [Press Release]. Retrieved from https://ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-reports-strong-third-quarter-2024-financial
3. PR Newswire. Harmony Biosciences Reports Strong Third Quarter 2024 Financial Results and Highlights Catalyst-Rich, Late-Stage Pipeline Poised to Deliver One or More New Launches Every Year Over Next Five Years [Press Release]. Retrieved from https://www.prnewswire.com/news-releases/harmony-biosciences-reports-strong-third-quarter-2024-financial-results-and-highlights-catalyst-rich-late-stage-pipeline-poised-to-deliver-one-or-more-new-launches-every-year-over-next-five-years-302289505.html
4. European Medicines Agency. Wakix, INN-pitolisant - European Medicines Agency [Assessment Report]. Retrieved from https://www.ema.europa.eu/en/documents/assessment-report/wakix-epar-public-assessment-report_en.pdf
5. Harmony Biosciences. Harmony Biosciences Reports Strong First Quarter Financial Results and Acceleration of Its Growth Strategy; Advances Pitolisant Franchise to Extend Revenue Potential Beyond 2040 [Press Release]. Retrieved from https://ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-reports-strong-first-quarter-financial