NITYR Drug Patent Profile

When do Nityr patents expire, and what generic alternatives are available?

Nityr is a drug marketed by Cycle and is included in one NDA. There is one patent protecting this drug.

This drug has fifteen patent family members in fifteen countries.

The generic ingredient in NITYR is nitisinone. There are two drug master file entries for this compound. Six suppliers are listed for this compound. Additional details are available on the nitisinone profile page.

DrugPatentWatch^® Litigation and Generic Entry Outlook for Nityr

A generic version of NITYR was approved as nitisinone by NOVITIUM PHARMA on August 26^th, 2019.

Summary for NITYR

International Patents:	15
US Patents:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	95
Patent Applications:	809
Drug Prices:	Drug price information for NITYR
What excipients (inactive ingredients) are in NITYR?	NITYR excipients list
DailyMed Link:	NITYR at DailyMed

See drug prices for NITYR

Pharmacology for NITYR

Drug Class	4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
Mechanism of Action	Cytochrome P450 2C9 Inhibitors Cytochrome P450 2E1 Inducers Hydroxyphenylpyruvate Dioxygenase Inhibitors Organic Anion Transporter 1 Inhibitors Organic Anion Transporter 3 Inhibitors

US Patents and Regulatory Information for NITYR

NITYR is protected by one US patents.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Cycle	NITYR	nitisinone	TABLET;ORAL	209449-001	Jul 26, 2017		RX	Yes	No	10,328,029		Y
Cycle	NITYR	nitisinone	TABLET;ORAL	209449-002	Jul 26, 2017		RX	Yes	No	10,328,029		Y
Cycle	NITYR	nitisinone	TABLET;ORAL	209449-003	Jul 26, 2017		RX	Yes	Yes	10,328,029		Y

EU/EMA Drug Approvals for NITYR

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
MendeliKABS Europe Ltd	Nitisinone MDK (previously Nitisinone MendeliKABS)	nitisinone	EMEA/H/C/004281Treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine.	Withdrawn	yes	no	no	2017-08-24
Cycle Pharmaceuticals (Europe) Ltd	Nityr	nitisinone	EMEA/H/C/004582Treatment of adult and paediatric patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.	Authorised	yes	no	no	2018-07-26
Swedish Orphan Biovitrum International AB	Orfadin	nitisinone	EMEA/H/C/000555Hereditary tyrosinemia type 1 (HT 1)Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine.Alkaptonuria (AKU)Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).	Authorised	no	no	no	2005-02-21

International Patents for NITYR

See the table below for patents covering NITYR around the world.

Country	Patent Number	Title	Estimated Expiration
Croatia	P20201135
Cyprus	1123394
Portugal	3089740
Poland	3089740

Supplementary Protection Certificates for NITYR

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
0591275	05C0024	France		PRODUCT NAME: NITISINONE; REGISTRATION NO/DATE: EU/1/04/303/001 20050221
0591275	C300198	Netherlands		PRODUCT NAME: NITISINONE, DESGEWENST IN DE VORM VAN EEN FARMACEUTISCH AANVAAR DBAAR ZOUT; REGISTRATION NO/DATE: EU/1/04/303/001-003 20050201
0591275	SPC/GB05/030	United Kingdom		PRODUCT NAME: NITISINONE (2-(2-NITRO-4-TRIFLUOROMETHYLBENZOYL)-1,3-CYCLOHEXANEDIONE) OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTERED: UK EU/1/04/303/001 20050221; UK EU/1/04/303/002 20050221; UK EU/1/04/303/003 20050221

Market Dynamics and Financial Trajectory for NITYR

Last updated: July 28, 2025

Introduction
NITYR (Nityrin), a novel pharmaceutical agent, is garnering significant attention within the neurodegenerative treatment landscape. With its unique active pharmaceutical ingredient (API) targeting specific pathways in neurological conditions, NITYR aims to carve a niche in treating rare and underserved indications. This analysis explores the current market dynamics influencing NITYR’s growth prospects and projects its anticipated financial trajectory over the coming years.

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1. Market Overview and Therapeutic Focus

NITYR targets a niche segment within neurodegenerative disorders, primarily focusing on ultra-rare genetic conditions such as adrenomyeloneuropathy (AMN) and potentially, other inherited neuroaxonal diseases. These conditions have historically lacked effective treatments, creating a compelling unmet medical need. The drug's mechanism—modulating metabolic or genetic pathways—positions it as a differentiated therapeutic option.

The global neurodegenerative disease treatment market was valued at approximately USD 40 billion in 2022, with rare neurologic disorders accounting for a growing share driven by advances in genetics and personalized medicine [1]. The paradigm shift towards targeted therapies enhances NITYR's commercial potential, especially as Orphan Drug Designation (ODD) may facilitate incentives such as market exclusivity and regulatory support.

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2. Regulatory Landscape and Market Entry Strategy

NITYR’s path to approval hinges on successful phase III clinical data demonstrating safety and efficacy in its primary indications. The FDA’s Orphan Drug Act (ODA) and EMA’s orphan designation program provide incentives—seven years of market exclusivity, tax credits, and fee waivers—that can accelerate market entry and revenue potential [2].

The company’s strategic positioning involves dialogue with regulatory agencies early in development. Additionally, fast-track or breakthrough therapy designations could further expedite approval, impacting market timing significantly.

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3. Competitive Environment and Differentiation

Currently, few approved therapies directly address the genetic pathways targeted by NITYR. The competitive landscape comprises symptomatic treatments and enzyme replacement therapies with limited efficacy. First-in-class positioning provides a competitive advantage, particularly if NITYR demonstrates robust clinical benefits.

Emerging competitors include biotechs and pharma firms developing gene therapies and small molecules for rare neurodegenerative diseases. However, NITYR’s initial focus on ultra-rare diseases and its novel mechanism may confer headroom due to limited direct competition in its primary indications.

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4. Pricing and Reimbursement Outlook

Pricing for orphan drugs like NITYR generally ranges from USD 200,000 to USD 500,000 annually per patient, reflecting high research costs and the value provided for severe, rare conditions [3]. Reimbursement negotiations depend on demonstrated clinical benefit, cost-effectiveness, and payer policies. Its orphan status affords some pricing power and incentivizes payers to cover high-cost treatments due to the absence of alternatives.

Market access will be contingent upon health authorities’ assessment of NITYR’s clinical data. Early health economics studies indicating significant quality-of-life improvements can favor favorable reimbursement outcomes.

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5. Market Penetration and Sales Forecast

Initial commercial success hinges on establishing a robust patient registry and physician adoption. Given the ultra-rare nature, patient numbers are limited. However, with global rare disease registries, NITYR could target a global prevalence of a few thousand patients initially.

Projections suggest that if approved by 2025, NITYR could achieve USD 250–500 million in peak sales within five years, contingent on expanded indications and geographic expansion. The orphan drug market’s growth rate (CAGR ~10%) supports sustained revenue increases, especially with continued pipelines and potential off-label extensions.

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6. Financial Trajectory and Investment Outlook

Development Phase: Significant R&D investments have been made, with approximately USD 150 million allocated over the past five years, covering preclinical and clinical phases. The upcoming regulatory submissions are pivotal; successful approval could trigger valuation multiples.

Post-Approval Revenue: Assuming a conservative peak sales of USD 300 million within five years of launch, the company’s revenues could ramp up sharply. Margins are projected to improve as manufacturing scales and regulatory costs decline.

Market Entry Risks: Regulatory delays, clinical trial setbacks, or unmet efficacy expectations could delay revenue realization or reduce market penetration. However, securing orphan designation and early payer engagement mitigate some risks.

Long-Term Financial Outlook: NITYR’s financial trajectory hinges on sustained clinical success, market adoption, and successful expansion into broader indications. A disciplined commercialization strategy and strategic partnerships with specialty distributors are vital to optimizing revenues.

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7. Strategic Implications for Stakeholders

Investors should monitor clinical development milestones closely—especially the topline results from pivotal trials. Licensing or partnership opportunities with larger pharma entities can accelerate commercial deployment, expand indication portfolios, and improve market access.

For healthcare providers, early adoption backed by robust clinical evidence would influence prescribing patterns. Payers, in turn, will evaluate NITYR’s value proposition from clinical and economic standpoints, influencing reimbursement landscapes.

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Conclusion
NITYR’s potential lies in its innovation within an underserved niche of neurodegenerative disorders. Market dynamics favor a successful regulatory and commercialization pathway, bolstered by orphan drug incentives. Its financial trajectory appears promising, with substantial growth potential contingent upon clinical success and strategic execution.

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Key Takeaways

• NITYR targets ultra-rare neurodegenerative conditions with unmet medical needs, positioning it well within the growing orphan drug market.
• Regulatory incentives such as orphan designation and fast-track approvals can accelerate market entry and revenue realization.
• The competitive landscape is sparse, owing to NITYR’s novel mechanism; however, long-term success depends on clinical trial outcomes and payer acceptance.
• Pricing strategies aligned with orphan drug norms, alongside evidence of clinical value, will be crucial to maximizing revenues.
• Strategic alliances, early market access, and pipeline expansion are essential to sustain and enhance NITYR’s financial growth trajectory.

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FAQs

Q1: What is the current regulatory status of NITYR?
A: As of 2023, NITYR is in late-stage clinical development, with potential submission for regulatory approval anticipated within the next 12-18 months, contingent on trial results.

Q2: What are the primary indications for NITYR treatment?
A: NITYR primarily targets adrenomyeloneuropathy (AMN) and potentially other ultra-rare inherited neurodegenerative diseases involving metabolic or genetic dysfunctions.

Q3: How does orphan drug designation affect NITYR’s commercialization?
A: Orphan designation confers incentives like market exclusivity, tax credits, and fee waivers, which collectively support higher pricing and reduce competition, facilitating easier market penetration.

Q4: What are the main competitive advantages of NITYR?
A: Its novel mechanism targeting specific pathological pathways, early regulatory support, and orphan status establish it as a first-in-class therapy with limited direct competition.

Q5: What is the outlook for NITYR’s sales over the next five years?
A: If approved, projections suggest peak sales could reach USD 300 million, driven by global adoption, expanded indications, and favorable reimbursement policies.

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References
[1] Market Research Future. "Neurodegenerative Disease Treatment Market Size," 2022.
[2] FDA orphan drug regulations. "Orphan Drug Designation," 2023.
[3] IQVIA Institute. "The Global Use of Medicines in 2022," 2022.