ORFADIN Drug Patent Profile

Which patents cover Orfadin, and what generic alternatives are available?

Orfadin is a drug marketed by Swedish Orphan and is included in two NDAs. There is one patent protecting this drug.

This drug has twenty-four patent family members in twenty-two countries.

The generic ingredient in ORFADIN 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 Orfadin

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

Summary for ORFADIN

International Patents:	24
US Patents:	1
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	125
Clinical Trials:	12
Patent Applications:	499
Drug Prices:	Drug price information for ORFADIN
What excipients (inactive ingredients) are in ORFADIN?	ORFADIN excipients list
DailyMed Link:	ORFADIN at DailyMed

See drug prices for ORFADIN

Recent Clinical Trials for ORFADIN

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

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

See all ORFADIN clinical trials

Pharmacology for ORFADIN

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 ORFADIN

ORFADIN 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
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-001	Jan 18, 2002	AB	RX	Yes	No
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-004	Jun 13, 2016	AB	RX	Yes	Yes
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-002	Jan 18, 2002	AB	RX	Yes	No
Swedish Orphan	ORFADIN	nitisinone	CAPSULE;ORAL	021232-003	Jan 18, 2002	AB	RX	Yes	No
Swedish Orphan	ORFADIN	nitisinone	SUSPENSION;ORAL	206356-001	Apr 22, 2016		RX	Yes	Yes	9,301,932		Y

Expired US Patents for ORFADIN

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 ORFADIN

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 ORFADIN

When does loss-of-exclusivity occur for ORFADIN?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

Chile

China

Croatia

Cyprus

Denmark

European Patent Office

Hungary

Israel

Japan

Mexico

New Zealand

Poland

Russian Federation

San Marino

Serbia

Slovenia

Spain

Ukraine

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering ORFADIN around the world.

Country	Patent Number	Title	Estimated Expiration
Romania	101429	COMPOZITIE ERBICIDA SOLIDA (SOLID HERBICIDE COMPOSITION)
Hungary	192443	HERBICIDE COMPOSITIONS CONTAINING 2-/2-SUBSTITUTED BENZOYL/-CYCLOHEXANE-1,3-DIONE DERIVATIVES AS ACTIVE AGENTS AND PROCESS FOR PRODUCING THE ACTIVE AGENTS
Japan	6038132
Norway	162663
Russian Federation	2014101990	ЖИДКАЯ ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ, ВКЛЮЧАЮЩАЯ НИТИЗИНОН
Australia	3266084
World Intellectual Property Organization (WIPO)	2012177214

Supplementary Protection Certificates for ORFADIN

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	CA 2005 00029	Denmark
0137963	97C0042	Belgium		PRODUCT NAME: 2-(2-BENZOYL-SUBSTITUE)-1,3-CYCLOHEXANE-DIONES; REGISTRATION NO/DATE: 8452/B 19930121
0186118	C350003	Netherlands		PRODUCT NAME: MESOTRIONE, DESGEWENST IN DE VORM VAN EEN ZOUT; REGISTRATION NO/DATE: 12204N 20010504
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	24/2005	Austria		PRODUCT NAME: NITISINON ODER EIN PHARMAZEUTISCH ZULAESSIGES SALZ HIERVON; REGISTRATION NO/DATE: EU/1/04/303/001- EU/1/04/303/003 20050221

ORFADIN Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Orfadin

Introduction to Orfadin

Orfadin, also known as nitisinone, is a medication used for the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder. It is manufactured by Swedish Orphan Biovitrum AB (publ), now known as Sobi.

Approval and Benefit/Risk Profile

Orfadin was approved by Health Canada in 2016 for the treatment of HT-1 in combination with dietary restrictions of tyrosine and phenylalanine. The approval was based on a favourable benefit/risk profile, as the drug helps manage the accumulation of toxic metabolites associated with the disease[4].

Pharmacoeconomic Analysis

The pharmacoeconomic analysis of Orfadin, conducted by CADTH, highlights the drug's cost-effectiveness. The analysis showed an incremental cost-utility ratio (ICUR) of $377,025 per quality-adjusted life year (QALY) gained when compared to best supportive care (BSC) alone. This analysis considered various scenarios, including different doses of nitisinone and the inclusion of diet costs and potential liver transplantation for treated patients[1].

Market Performance

Revenue Trends

Orfadin's revenue has seen fluctuations in recent years. In the third quarter of 2021, Orfadin sales were SEK 116 million, which represented a 25% decrease compared to the same period in 2020, largely due to generic competition and associated price erosion. Year-to-date sales for 2021 were SEK 338 million, a 35% decrease from the previous year[2].

Geographic Distribution

Sobi's revenue, including that from Orfadin, is distributed across various geographic regions. Europe and North America are significant markets, with the United States now being the largest revenue driver for Sobi. International expansion, including launches in Japan, Latin America, and South Korea, has also contributed to the company's growth[3].

Competitive Landscape

Orfadin faces competition from generic versions, which has impacted its sales negatively. The introduction of generic alternatives has led to price erosion, affecting the drug's revenue. However, Sobi's diversified portfolio and strong presence in the rare disease market help mitigate this impact[2].

Financial Impact on Sobi

Revenue Contribution

Orfadin is part of Sobi's Specialty Care portfolio, which includes other medicines like Tegsedi and Gamifant. While Orfadin's sales have declined due to generic competition, the overall Specialty Care revenue has been supported by the growth of other products in the portfolio. For instance, Gamifant saw a significant increase in sales, driven by increased recognition and physician experience[3].

Gross Margin and Operating Expenses

Sobi's gross margin has remained relatively stable, with a slight decrease due to factors like mandatory price reductions and the high share of low-margin sales. Operating expenses, including selling and administrative costs, have increased, but the company has maintained a strong EBITA margin, indicating robust profitability[2].

Strategic Outlook

Sobi has diversified its portfolio significantly over the past few years, reducing its dependence on any single product. The company expects a majority of its revenue to come from medicines launched after 2019 by 2026. This strategic diversification helps Sobi navigate market challenges, including competition from generics[3].

Research and Development

Sobi continues to advance its R&D pipeline, which is crucial for maintaining market competitiveness. New clinical data and regulatory filings, particularly for drugs like Gamifant and efanesoctocog alfa, are key to the company's future growth. This focus on R&D ensures a steady stream of innovative products to replace or complement existing ones like Orfadin[3].

Sustainability and Patient Impact

Sobi's commitment to improving the lives of patients with rare diseases remains a core part of its strategy. With only 5% of rare diseases having approved medicines, Sobi's work in this area is critical. The company's financial performance is closely tied to its ability to deliver innovative treatments that transform patient lives[3].

Key Takeaways

• Orfadin's approval and pharmacoeconomic analysis highlight its effectiveness in treating HT-1.
• The drug faces significant competition from generics, impacting its revenue.
• Sobi's diversified portfolio and strong R&D pipeline support the company's financial stability and growth.
• Geographic expansion and new product launches are key to Sobi's future success.
• The company remains committed to improving the lives of patients with rare diseases.

Frequently Asked Questions (FAQs)

What is Orfadin used for?

Orfadin (nitisinone) is used for the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder, in combination with dietary restrictions of tyrosine and phenylalanine[4].

How has generic competition affected Orfadin's sales?

Generic competition has significantly impacted Orfadin's sales, leading to a decline in revenue due to price erosion and increased competition in the market[2].

What is Sobi's strategy to mitigate the impact of generic competition on Orfadin?

Sobi has diversified its product portfolio to reduce dependence on any single product. The company focuses on launching new medicines and expanding its presence in various geographic regions[3].

How does Orfadin's pharmacoeconomic analysis impact its market dynamics?

The pharmacoeconomic analysis shows that Orfadin is cost-effective, with an ICUR of $377,025 per QALY gained. This analysis supports the drug's value in treating HT-1, despite its high cost[1].

What role does R&D play in Sobi's financial trajectory?

Sobi's R&D efforts are crucial for maintaining market competitiveness and ensuring future growth. New clinical data and regulatory filings for innovative products help the company stay ahead in the rare disease market[3].