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 three 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-eight 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:	38
US Patents:	2
Tradenames:	3
Applicants:	6
NDAs:	7
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	6
Raw Ingredient (Bulk) Api Vendors:	125
Clinical Trials:	16
Patent Applications:	853
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
Parexel	Phase 1
Cycle Pharmaceuticals Ltd.	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
Torrent	NITISINONE	nitisinone	CAPSULE;ORAL	215908-004	Jan 9, 2023		DISCN	No	No
Torrent	NITISINONE	nitisinone	CAPSULE;ORAL	215908-003	Jan 9, 2023		DISCN	No	No
Eton	NITISINONE	nitisinone	CAPSULE;ORAL	216201-004	May 25, 2023	AB	RX	No	No
Cycle	NITYR	nitisinone	TABLET;ORAL	209449-001	Jul 26, 2017		RX	Yes	No	10,328,029		Y
Torrent	NITISINONE	nitisinone	CAPSULE;ORAL	215908-002	Jan 9, 2023		DISCN	No	No
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002	AB	RX	Yes	No
Medunik	NITISINONE	nitisinone	CAPSULE;ORAL	212390-001	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	021232-002	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	021232-001	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/004281 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.	Withdrawn	yes	no	no	2017-08-24
Cycle Pharmaceuticals (Europe) Ltd	Nityr	nitisinone	EMEA/H/C/004582 Treatment 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/000555 Hereditary 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
Slovenia	3089740
Serbia	60551	FARMACEUTSKA KOMPOZICIJA (PHARMACEUTICAL COMPOSITION)
Denmark	3089740
Portugal	3089740
Russian Federation	2014101990	ЖИДКАЯ ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ, ВКЛЮЧАЮЩАЯ НИТИЗИНОН
San Marino	T201600097	COMPOSIZIONE FARMACEUTICA LIQUIDA CHE COMPRENDE NITISONE
European Patent Office	3089740	COMPOSITION PHARMACEUTIQUE (PHARMACEUTICAL COMPOSITION)

Supplementary Protection Certificates for nitisinone

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

Nitisinone Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Nitisinone

Introduction to Nitisinone

Nitisinone is a medication primarily used to treat hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder. The drug has been gaining traction due to its efficacy and the increasing awareness of rare diseases.

Global Market Size and Growth

The global Nitisinone market is projected to experience significant growth. As of 2024, the market size is estimated to be USD 101.5 million and is expected to expand to USD 205.47 million by 2031, with a Compound Annual Growth Rate (CAGR) of 10.60% from 2024 to 2031[1].

Key Drivers of Market Growth

Several factors are driving the growth of the Nitisinone market:

Rising Prevalence of Rare Genetic Disorders

The increasing prevalence of HT-1 and other rare genetic disorders is a major driver. Early diagnosis and treatment initiatives are contributing to the demand for nitisinone[1].

Increased Awareness and Early Diagnosis

Advancements in diagnostic tools and newborn screening programs have improved the early identification and treatment of HT-1, boosting the market[1].

Supportive Government Incentives

Government incentives for orphan drugs have been instrumental in promoting the development and use of nitisinone. These incentives encourage pharmaceutical companies to invest in rare disease treatments[1].

Technological Advancements

Improvements in healthcare infrastructure, particularly in emerging markets, and technological advancements in diagnostic tools are also driving market growth[1].

Regional Market Dynamics

North America

North America currently dominates the Nitisinone market, driven by a high prevalence of HT-1, advanced healthcare infrastructure, and strong awareness of rare diseases. The region's comprehensive diagnostic programs and robust healthcare policies further support this dominance[1].

Asia Pacific

The Asia Pacific region is growing at the fastest CAGR, driven by improving healthcare infrastructure, increased awareness of rare diseases, and expanding access to diagnostic and treatment services. Countries like China, India, and Japan are key contributors to this growth[1].

Latin America and Middle East & Africa

Latin America and the Middle East & Africa also show potential for growth, although at a slower pace compared to North America and the Asia Pacific. These regions are expected to grow at CAGRs of 10.0% and 10.3%, respectively, from 2024 to 2031[1].

Financial Performance of Key Players

Eton Pharmaceuticals

Eton Pharmaceuticals, a company involved in the rare disease treatment market, has reported significant financial growth. In Q4 2023, they achieved a 109% year-over-year increase in product sales and royalty revenue, reaching $7.3 million. This growth is partly attributed to the success of their products like ALKINDI SPRINKLE and Carglumic Acid, which are used in treating rare conditions[2][5].

Sobi and Cycle Pharmaceuticals

Other key players in the Nitisinone market include Sobi and Cycle Pharmaceuticals. These companies are major contributors to the market's financial trajectory through their investments in research and development, and their efforts in expanding treatment availability for HT-1[4].

Challenges and Restraints

High Cost of Nitisinone

One of the significant challenges facing the Nitisinone market is the high cost of the drug. This can limit accessibility, especially in regions with less robust healthcare systems[1].

Regulatory Challenges

Regulatory hurdles can also restrain market expansion. The approval process for orphan drugs can be complex and time-consuming, affecting the timely availability of nitisinone in various markets[1].

Recent Developments and Innovations

Generic Nitisinone Capsules

Analog Pharma and Dipharma have recently received FDA approval for their 20 mg generic nitisinone capsules, which are stable at room temperature for 3 years. This development is expected to improve patient access and compliance[3].

Pipeline Developments

Companies like Eton Pharmaceuticals are making significant strides in their pipelines. For example, Eton's ET-400, a proprietary formulation of oral hydrocortisone, has passed its pivotal bioequivalence study and is on track for NDA submission in Q2 2024[2][5].

Market Trends

Personalized Medicine

There is a growing emphasis on personalized medicine, which includes tailored treatments for rare genetic disorders. This trend is expected to drive innovation and demand for nitisinone[1].

Expanding Healthcare Infrastructure

Improving healthcare infrastructure in emerging markets is another trend that supports the growth of the Nitisinone market. This expansion increases access to diagnostic and treatment services for patients with HT-1[1].

Increased Investment in R&D

Increased investment in research and development for rare diseases is a significant trend. This investment is crucial for developing new treatments and improving existing ones, such as nitisinone[1].

Key Takeaways

• The global Nitisinone market is expected to grow significantly, reaching USD 205.47 million by 2031.
• Key drivers include the rising prevalence of HT-1, increased awareness and early diagnosis, and supportive government incentives.
• North America dominates the market, but the Asia Pacific region is growing at the fastest CAGR.
• High costs and regulatory challenges are significant restraints.
• Recent developments, such as the approval of generic nitisinone capsules and pipeline advancements, are improving market dynamics.

FAQs

What is the current global market size for Nitisinone?

The global Nitisinone market size is estimated to be USD 101.5 million in 2024[1].

What is the projected CAGR for the Nitisinone market from 2024 to 2031?

The Nitisinone market is expected to grow at a CAGR of 10.60% from 2024 to 2031[1].

Which region dominates the Nitisinone market?

North America currently dominates the Nitisinone market due to its high prevalence of HT-1 and advanced healthcare infrastructure[1].

What are the main challenges facing the Nitisinone market?

The high cost of nitisinone and regulatory challenges are significant restraints to market expansion[1].

Which companies are key players in the Nitisinone market?

Key players include Sobi, Cycle Pharmaceuticals, and companies like Eton Pharmaceuticals that are involved in rare disease treatments[2][4].

Cited Sources:

1. Cognitive Market Research: Global Nitisinone Market Report 2024.
2. GlobeNewswire: Eton Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results.
3. BioSpace: Analog Pharma and Dipharma announce US FDA approval of abbreviated new drug application (ANDA) of 20 mg generic Nitisinone capsules.
4. Valuates Reports: Global Nitisinone Market Research Report 2024.
5. Eton Pharmaceuticals: Eton Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results.