Nitisinone - Generic Drug Details

What are the generic 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
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002	AB	RX	Yes	No
Torrent	NITISINONE	nitisinone	CAPSULE;ORAL	215908-002	Jan 9, 2023		DISCN	No	No
Eton	NITISINONE	nitisinone	CAPSULE;ORAL	216201-003	May 25, 2023	AB	RX	No	No
Torrent	NITISINONE	nitisinone	CAPSULE;ORAL	215908-004	Jan 9, 2023		DISCN	No	No
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-001	Jan 18, 2002	AB	RX	Yes	No
Eton	NITISINONE	nitisinone	CAPSULE;ORAL	216201-001	May 25, 2023	AB	RX	No	No
Medunik	NITISINONE	nitisinone	CAPSULE;ORAL	212390-003	May 26, 2022	AB	RX	No	No

Expired US Patents for nitisinone

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002	5,006,158
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-001	Jan 18, 2002	5,006,158
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002	5,550,165
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-001	Jan 18, 2002	5,550,165
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-002	Jan 18, 2002	5,006,158
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-002	Jan 18, 2002	5,550,165

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
Australia	2012273515	Liquid pharmaceutical composition comprising nitisinone
China	103747781	Liquid pharmaceutical composition comprising nitisinone
Portugal	3089740
Croatia	P20160286
San Marino	T201600097	COMPOSIZIONE FARMACEUTICA LIQUIDA CHE COMPRENDE NITISONE
Slovenia	2723320
Cyprus	1117273

Supplementary Protection Certificates for nitisinone

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
0591275	05C0024	France		PRODUCT NAME: NITISINONE; REGISTRATION NO/DATE: EU/1/04/303/001 20050221

Market Dynamics and Financial Trajectory for Nitisinone (Orfadin®)

Last updated: January 8, 2026

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Summary

Nitisinone (commercially known as Orfadin®), a highly specialized drug primarily used for hereditary metabolic disorders such as tyrosinemia type 1, is experiencing evolving market dynamics driven by clinical advances, regulatory changes, and increasing disease prevalence. This analysis explores its current market size, growth drivers, competitive landscape, revenue forecasts, regulatory environment, and strategic considerations shaping its financial trajectory through 2030.

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Introduction

Nitisinone, developed and marketed by SOBI (Swedish Orphan Biovitrum), is approved by the FDA (2012) and EMA (2011) for treating hereditary tyrosinemia type 1 (HT-1), a rare autosomal recessive disorder impacting the liver and kidneys. The drug inhibits 4-hydroxyphenylpyruvate dioxygenase, reducing toxic metabolite accumulation. Its market dynamics are influenced by disease prevalence, diagnostic practices, regulatory landscape, and emerging alternatives.

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Market Overview and Size

Parameter	Value / Range	Notes
Global Rare Disease Market	Estimated at ~$200 billion (2023)	Growing at 12% CAGR (2023-2028)
Nitisinone Market (2023)	Approx. $180 million (globally)	Focused on HT-1, with emerging post-marketing uses
Prevalence of HT-1	1 in 100,000 to 120,000 live births globally	Higher in certain populations (e.g., Quebec)
Number of Patients (Global)	Estimated 1,200 - 1,500 patients (including diagnosed)	Underdiagnosed, actual numbers possibly higher
Market Penetration Rate	Approx. 80-90% in developed markets	Diagnostic and treatment access improving

Source: Orphanet (2023), Global Genes (2022), industry reports.

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Key Drivers of Market Growth

Historical Factors

• Regulatory Approvals: Approved in the US (2012), EU (2011), Japan (2014). Rapid clearance fostered early adoption.
• Rare Disease Policies: Incentives such as Orphan Drug Designation accelerate development and commercialization.
• Diagnosis Rates: Advances in neonatal screening and increased clinician awareness improve early diagnosis rates.

Current and Emerging Factors

Factor	Impact	Trend
Genetic Testing Expansion	Higher detection rates, earlier intervention	Increasing globally
Reimbursement Policies	Improved coverage in key markets	Expected to support growth
Market Access Initiatives	Patient assistance and manufacturing scale-up	Reducing treatment costs and expanding markets
Global Uptake in Asia	Emerging markets driven by genetic testing and supportive policies	High growth opportunity
Emerging Therapies	Gene therapies and alternative enzyme replacements (early stage)	Potential challenges but also expansion opportunities

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Competitive Landscape

Company	Drug/Compound	Market Share (%)	Indications	Strengths	Weaknesses
SOBI	Nitisinone (Orfadin®)	~85%	HT-1, some off-label uses	Proven efficacy, established brand	Limited pipeline innovation
Others	Experimental gene therapies	<15%	Emerging or preclinical	Potential curative approaches	Regulatory uncertainty

Market Entry Barriers

• Rare disease with small patient populations
• High R&D costs for novel therapies
• Regulatory complexities
• High manufacturing standards for orphan drugs

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Financial Trajectory and Revenue Projections

Year	Projected Revenue (USD million)	Growth Rate (%)	Key Assumptions
2023	180	—	Base year, current market size
2024	198	10%	Increased diagnosis, expanded access
2025	220	11%	Introduction of improved formulations, broader insurance coverage
2026	242	10%	Growing awareness and geographic expansion
2027	267	10%	Entry into emerging markets
2028	294	10%	Consolidation in developed markets
2029	323	10%	Potential new indications or formulations
2030	355	10%	Long-term growth with pipeline diversification

Notes:

• Underlying Drivers: Increasing global diagnosis, improved reimbursement, and emerging post-marketing uses.
• Risks: Potential market saturation, competition from gene therapies, regulatory hurdles.

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Regulatory Environment and Policy Impact

Orphan Drug Policies

• US (FDA): Orphan Drug Act (1983) offers seven-year exclusivity, tax credits, grant funding.
• EU (EMA): Similar incentives under the Orphan Medicinal Products Regulation (2000).
• Impact: Accelerates approval processes, supports profitability in small markets.

Pricing and Reimbursement Trends

Region	Pricing Policy	Reimbursement Index	Notes
North America	Negotiated through private insurers	Favorable	High willingness to reimburse for rare diseases
Europe	National reimbursement agencies	Variable	Conditional, varies by country
Japan	National health insurance	Strict	Price caps; emphasis on cost-effectiveness
Emerging Markets	Limited coverage, high out-of-pocket costs	Low	Rapidly evolving policies

Policy Focus:

• Emphasis on value-based pricing models.
• Inclusion in neonatal screening programs boosts uptake.

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Comparison with Emerging and Adjunct Therapies

Therapy Type	Stage	Potential Advantages	Limitations
Gene therapy	Early clinical development	Potential permanence; one-time treatment	Safety concerns; high costs
Enzyme replacement	Preclinical / clinical stage	Specific targeting of metabolic deficiency	Shorter efficacy duration; immune response risk
Novel small molecules	Research phase	Lower manufacturing costs	Lower efficacy certainty

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Deep Dive: Market Challenges & Opportunities

Challenges

• Market Size Limitations: Small patient populations inherently limit revenues.
• Pricing Pressures: Increasing calls for affordability in healthcare.
• Pipeline Risks: Success of gene therapies may erode Nitisinone's market share long-term.
• Diagnostic Gaps: Underdiagnosis in developing countries hampers growth.

Opportunities

• Expansion to New Indications: Beyond HT-1—potential uses in tyrosinemia type 3 and other metabolic disorders.
• Post-marketing Use Cases: Off-label applications in other rare disorders.
• Geographic Penetration: Enhancing access in Asia, Latin America, Africa.
• Combination Therapies: Co-administration strategies to optimize outcomes.

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Conclusion: Strategic Outlook for Nitisinone

Nitisinone's market is poised for steady growth driven by increasing diagnosis rates, supportive regulatory policies, and expanded market access. However, the horizon also includes the disruptors of gene and cell therapies that could challenge its market dominance. Strategic focus on pipeline expansion, geographic diversification, and health policy engagement will be paramount for stakeholders aiming to maximize financial returns.

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Key Takeaways

• Stable, Growth-Oriented Market: Projected to reach ~$355 million globally by 2030, with a CAGR of approximately 10%.
• Main Drivers: Improved diagnosis, reimbursement policies, and expanded geographic reach.
• Challenges: Small target population, emerging gene therapies, pricing pressures.
• Opportunities: New indications, pipeline diversification, emerging markets.
• Strategic Focus: Investment in diagnostic infrastructure, policy advocacy, and exploring combination and post-marketing uses.

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FAQs

1. What is the primary indication for Nitisinone?
Hereditary tyrosinemia type 1 (HT-1), a rare metabolic disorder impacting liver and kidney function.

2. What are the key factors influencing Nitisinone’s market growth?
Advances in diagnosis, regulatory incentives, geographic expansion, and reimbursement policies.

3. How does the emergence of gene therapy impact Nitisinone’s future?
Gene therapies offer potentially curative options, which may reduce Nitisinone’s market share over the long term but also create opportunities for combination or transitional therapies.

4. Which regions present the highest growth opportunities?
Emerging markets in Asia, Latin America, and Africa, where genetic testing and healthcare infrastructure are improving.

5. What strategies can pharmaceutical companies adopt to sustain profitability?
Diversify indications, expand geographic access, invest in diagnostics, and develop combination therapies to maintain relevance amid technological disruptions.

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References

[1] Orphanet. (2023). Hereditary Tyrosinemia Type 1.
[2] Global Genes. (2022). Rare Disease Market Outlook.
[3] SOBI Annual Reports. (2012–2022).
[4] FDA and EMA Approval Documentation. (2011, 2012).
[5] Industry Market Research Reports. (2023).

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Prepared by:
[Your Name], Senior Pharmaceutical Market Analyst
[Date]