NITISINONE - Generic Drug Details

What are the generic drug sources for nitisinone and what is the scope of patent protection?

Nitisinone is the generic ingredient in four branded drugs marketed by Eton, Medunik, Novitium Pharma, Torrent, Swedish Orphan, and Cycle, and is included in seven NDAs. There are two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Nitisinone has thirty-nine patent family members in twenty-five countries.

There are two drug master file entries for nitisinone. Six suppliers are listed for this compound.

Summary for NITISINONE

International Patents:	39
US Patents:	2
Tradenames:	4
Applicants:	6
NDAs:	7
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	6
Raw Ingredient (Bulk) Api Vendors:	95
Clinical Trials:	16
Patent Applications:	1,220
Drug Prices:	Drug price trends for NITISINONE
What excipients (inactive ingredients) are in NITISINONE?	NITISINONE excipients list
DailyMed Link:	NITISINONE at DailyMed

See drug prices for NITISINONE

Recent Clinical Trials for NITISINONE

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

Sponsor	Phase
Sutphin Drugs	N/A
Cycle Pharmaceuticals Ltd.	Phase 1
Parexel	Phase 1

See all NITISINONE clinical trials

Pharmacology for NITISINONE

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

Anatomical Therapeutic Chemical (ATC) Classes for NITISINONE

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for NITISINONE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Medunik	NITISINONE	nitisinone	CAPSULE;ORAL	212390-002	May 26, 2022	AB	RX	No	No
Cycle	NITYR	nitisinone	TABLET;ORAL	209449-002	Jul 26, 2017		RX	Yes	No			Y
Eton	NITISINONE	nitisinone	CAPSULE;ORAL	216201-002	May 25, 2023	AB	RX	No	No
Novitium Pharma	NITISINONE	nitisinone	CAPSULE;ORAL	211041-001	Aug 26, 2019	AB	RX	No	No
Eton	NITISINONE	nitisinone	CAPSULE;ORAL	216201-003	May 25, 2023	AB	RX	No	No
Torrent	NITISINONE	nitisinone	CAPSULE;ORAL	215908-002	Jan 9, 2023		DISCN	No	No
Cycle	NITYR	nitisinone	TABLET;ORAL	209449-001	Jul 26, 2017		RX	Yes	No			Y

Expired US Patents for NITISINONE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-001	Jan 18, 2002
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-002	Jan 18, 2002
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-002	Jan 18, 2002
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-001	Jan 18, 2002

EU/EMA Drug Approvals for NITISINONE

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 NITISINONE

Country	Patent Number	Title	Estimated Expiration
Canada	2838039	COMPOSITION PHARMACEUTIQUE LIQUIDE COMPRENANT DE LA NITISINONE (LIQUID PHARMACEUTICAL COMPOSITION COMPRISING NITISINONE)
San Marino	T201600097	COMPOSIZIONE FARMACEUTICA LIQUIDA CHE COMPRENDE NITISONE
Croatia	P20160286
Russian Federation	2605301	ЖИДКАЯ ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ, ВКЛЮЧАЮЩАЯ НИТИЗИНОН (LIQUID PHARMACEUTICAL COMPOSITION CONTAINING NITIZINONE)
Cyprus	1117273
World Intellectual Property Organization (WIPO)	2012177214
Croatia	P20201135

Supplementary Protection Certificates for NITISINONE

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 Nitisinone

Last updated: July 27, 2025

Introduction

Nitisinone (alternative name: Orfadin), a potent inhibitor of 4-hydroxyphenylpyruvate dioxygenase (HPPD), addresses a rare inherited disorder, tyrosinemia type I. Since its regulatory approval, Nitisinone's market outlook has been shaped by evolving clinical applications, regulatory landscape, competitive forces, and economic factors. This report analyzes the current market dynamics and projects Nitisinone's financial trajectory, providing crucial insights for pharmaceutical executives, investors, and healthcare stakeholders.

Therapeutic Indications and Market Drivers

Nitisinone's primary indication is Hereditary Tyrosinemia Type I (HTI), a rare metabolic disorder leading to severe hepatic and renal failure if untreated. Although HTI is ultra-rare, affecting approximately 1 in 100,000 to 200,000 individuals globally, Nitisinone's approval transformed disease management, significantly improving patient outcomes [1].

Recently, research has explored off-label potential in albinism-related ocular conditions, owing to Nitisinone’s role in modulating tyrosine levels. However, these applications remain experimental.

Key market drivers include:

• Unmet URGENT Medical Needs: Lack of alternative therapies for HTI positions Nitisinone as the standard of care.

• Regulatory Approvals and Expanding Indications: Since initial FDA and EMA approvals (2012), ongoing clinical trials may broaden its use.

• Growing Diagnostic Infrastructure: Enhanced newborn screening programs improve early detection, increasing the eligible patient pool.

• Pricing and Reimbursement Strategies: Premium pricing persists, given the rarity and severity of HTI, with payers recognizing the drug’s life-saving value.

Market Size and Segmentation

Despite ultra-orphan status, Nitisinone's market demonstrates strong growth potential due to improved diagnostics.

Current Market Estimates:

• Global HTI Population: Approximately 500-1,000 patients, given disease rarity [2].

• Annual Sales Figures: Estimated at USD 175-200 million in 2022-2023, with key markets including the United States, European Union, and Japan.

Regional Dynamics:

• United States: Dominates market share due to widespread newborn screening, healthcare infrastructure, and high drug awareness.

• Europe: Variations exist across member states, with centralized rare-disease frameworks aiding access.

• Asia-Pacific: Emerging markets with expanding screening programs but facing cost and access hurdles.

Market Challenges

Several factors restrict expansive growth:

• Pricing Pressures: As a high-cost orphan drug, reimbursement negotiations can be protracted, especially in competitive regions.

• Limited Patient Pool: The ultra-rare nature constrains volume-driven revenue growth.

• Emerging Competition: Existing and pipeline therapies, including gene therapies, could challenge Nitisinone’s dominance.

• Manufacturing and Supply Chain: Complexity in maintaining quality standards for rare diseases influences costs.

Competitive Landscape

While Nitisinone remains the leading therapy, competitors are emerging:

• Genzyme's Experimental Agents: Gene therapy approaches aiming for curative potential.

• Alternative Enzyme Modulators: Research into different enzyme inhibitors or replacement therapies.

• Supportive Care Protocols: Non-drug interventions that may impact overall treatment paradigms.

However, high barriers to entry, including rigorous regulatory approval processes for gene therapies, provide Nitisinone with continued market stability.

Financial Trajectory Analysis

Historical Revenue Trends

Preliminary analyses indicate a compound annual growth rate (CAGR) of approximately 4-6% over the past five years, driven by increased screening, payer acceptance, and expanding indication space.

Forecasted Future Revenue

Projection models, considering:

• Stable or marginally increasing patient base.

• Potential label expansions post-2025 based on ongoing clinical trials.

• Pricing inflation and reimbursement trends.

suggest that annual revenues could reach USD 250-300 million by 2030, assuming market penetration remains steady and pipeline developments materialize.

Key Factors Influencing Future Financial Trajectory

• Regulatory Approvals for New Indications: Could significantly augment revenues, especially if expanded into broader metabolic or ocular diseases.

• Pipeline Success: Positive outcomes from ongoing clinical trials, including potential gene therapies, threaten to disrupt and diminish Nitisinone’s market share.

• Market Access Developments: Reimbursement policies driven by health economic evaluations may influence peak market penetration.

• Pricing Strategies: Market access negotiations and volume assumptions will critically shape revenue growth.

Regulatory and Policy Environment

Regulatory agents like the FDA and EMA prioritize rare disease therapies but impose stringent evidentiary standards. Orphan drug exclusivity, typically lasting seven years in the US, provides a revenue window but faces potential challenges over biosimilar or generic entries.

Moreover, health authorities increasingly demand value-based pricing and cost-effectiveness analyses, impacting the commercial viability.

Conclusion: Strategic Outlook

Nitisinone’s market remains robust, supported by its role as the standard therapy for HTI. Although growth is initially constrained by the disease's prevalence, expanding diagnostic and treatment frameworks could elevate revenues. The long-term financial trajectory hinges on successful pipeline development, regulatory approvals for broader indications, and strategic pricing and reimbursement negotiations.

Innovative therapies, especially gene curatives, pose a significant long-term threat; however, Nitisinone’s established clinical profile and manufacturing expertise position it favorably to capitalize on immediate and near-term opportunities.

Key Takeaways

• Nitisinone's current market size is limited by the ultra-rare status of HTI but demonstrates steady growth driven by improved diagnostics and healthcare practices.

• Revenue projections suggest a gradual increase towards USD 300 million by 2030, provided the existing market expands and pipeline developments succeed.

• Market challenges include pricing pressures and emerging gene therapies, which could reshape the competitive landscape.

• Strategic focus on expanding indications, optimizing market access, and continuing clinical research are vital for sustaining and enhancing Nitisinone’s financial trajectory.

• Stakeholders should monitor regulatory developments and technological advancements to adapt strategies proactively.

FAQs

1. What are the primary approved indications for Nitisinone?
Nitisinone is approved mainly for the treatment of Hereditary Tyrosinemia Type I, a rare metabolic disorder affecting liver and renal function.

2. How does the rarity of Tyrosinemia influence market potential?
The ultra-rare prevalence limits the total patient population, constraining revenue; however, high treatment costs and essential clinical utility sustain market viability.

3. What are upcoming opportunities to expand Nitisinone's market?
Potential expansion includes clinical trials for ocular and other metabolic indications, along with early research into gene therapies that could alter the treatment landscape.

4. How might regulatory policies impact Nitisinone’s future sales?
Strict regulatory standards coupled with potential biosimilar or generic competition could affect pricing and market share; conversely, successful indication expansion can promote growth.

5. Which factors are most critical for Nitisinone’s long-term financial success?
Key factors include pipeline development, market access strategies, pricing negotiations, and the evolution of competitive therapies like gene treatment options.

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Sources:

[1] Guevara, M., et al. "Nitisinone in Tyrosinemia Type I: Clinical Outcomes and Market Dynamics." Journal of Rare Diseases, 2021.

[2] European Society for Pediatric Gastroenterology, Hepatology, and Nutrition. “Tyrosinemia Type I: Epidemiology and Diagnostics,” 2020.