SAPROPTERIN DIHYDROCHLORIDE - Generic Drug Details

What are the generic drug sources for sapropterin dihydrochloride and what is the scope of freedom to operate?

Sapropterin dihydrochloride is the generic ingredient in two branded drugs marketed by Biomarin Pharm, Annora Pharma, Dr Reddys, and Endo Operations, and is included in ten NDAs. There are ten patents protecting this compound and three Paragraph IV challenges. Additional information is available in the individual branded drug profile pages.

Sapropterin dihydrochloride has one hundred and seventeen patent family members in twenty-six countries.

There is one drug master file entry for sapropterin dihydrochloride. Five suppliers are listed for this compound.

Summary for SAPROPTERIN DIHYDROCHLORIDE

International Patents:	117
US Patents:	10
Tradenames:	2
Applicants:	4
NDAs:	10
Drug Master File Entries:	1
Finished Product Suppliers / Packagers:	5
Raw Ingredient (Bulk) Api Vendors:	64
Clinical Trials:	38
Patent Applications:	277
Drug Prices:	Drug price trends for SAPROPTERIN DIHYDROCHLORIDE
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for SAPROPTERIN DIHYDROCHLORIDE
What excipients (inactive ingredients) are in SAPROPTERIN DIHYDROCHLORIDE?	SAPROPTERIN DIHYDROCHLORIDE excipients list
DailyMed Link:	SAPROPTERIN DIHYDROCHLORIDE at DailyMed

See drug prices for SAPROPTERIN DIHYDROCHLORIDE

Recent Clinical Trials for SAPROPTERIN DIHYDROCHLORIDE

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

Sponsor	Phase
West China Hospital	Phase 1
University of Pittsburgh	Phase 1/Phase 2
Ira Fox	Phase 1/Phase 2

See all SAPROPTERIN DIHYDROCHLORIDE clinical trials

Pharmacology for SAPROPTERIN DIHYDROCHLORIDE

Drug Class	Phenylalanine Hydroxylase Activator
Mechanism of Action	Breast Cancer Resistance Protein Inhibitors P-Glycoprotein Inhibitors Phenylalanine Hydroxylase Activators

Anatomical Therapeutic Chemical (ATC) Classes for SAPROPTERIN DIHYDROCHLORIDE

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

Paragraph IV (Patent) Challenges for SAPROPTERIN DIHYDROCHLORIDE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
KUVAN	Powder for Oral Solution	sapropterin dihydrochloride	500 mg per packet	205065	1	2017-02-23
KUVAN	Powder for Oral Solution	sapropterin dihydrochloride	100 mg per packet	205065	1	2015-11-09
KUVAN	Tablets	sapropterin dihydrochloride	100 mg	022181	1	2014-06-05

US Patents and Regulatory Information for SAPROPTERIN DIHYDROCHLORIDE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Dr Reddys	SAPROPTERIN DIHYDROCHLORIDE	sapropterin dihydrochloride	POWDER;ORAL	209452-001	Mar 30, 2021	AB	RX	No	No
Annora Pharma	SAPROPTERIN DIHYDROCHLORIDE	sapropterin dihydrochloride	POWDER;ORAL	215420-002	Aug 18, 2022	AB	RX	No	No
Biomarin Pharm	KUVAN	sapropterin dihydrochloride	POWDER;ORAL	205065-001	Dec 19, 2013	AB	RX	Yes	Yes	7,566,714*PED				Y
Biomarin Pharm	KUVAN	sapropterin dihydrochloride	TABLET;ORAL	022181-001	Dec 13, 2007	AB	RX	Yes	Yes	7,612,073*PED				Y
Annora Pharma	SAPROPTERIN DIHYDROCHLORIDE	sapropterin dihydrochloride	POWDER;ORAL	215420-001	Aug 18, 2022	AB	RX	No	No

International Patents for SAPROPTERIN DIHYDROCHLORIDE

Country	Patent Number	Title	Estimated Expiration
Australia	2004311544	Crystalline forms of (6R) -L-erythro-tetrahydrobiopterin dihydrochloride
Malaysia	142120	STABLE TABLET FORMULATION OF TETRAHYDROBIOPTERIN
European Patent Office	1687308	Procédé pour la préparation de la forme cristalline B de (6R)-L-érythrotetrahydrobioptérin hydrochloride a partir d'autres formes cristallines (Process for preparating (6R)-L-erythrotetrahydrobiopterin hydrochloride crystalline form B from other crystalline forms)
South Korea	20160064248	테트라하이드로바이오프테린 투여 방법, 관련 조성물 및 측정 방법 (METHODS OF ADMINISTERING TETRAHYDROBIOPTERIN, ASSOCIATED COMPOSITIONS, AND METHODS OF MEASURING)
Croatia	P20210239

SAPROPTERIN DIHYDROCHLORIDE Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Sapropterin Dihydrochloride

Introduction to Sapropterin Dihydrochloride

Sapropterin dihydrochloride, commonly known by the brand name Kuvan, is a medication used to treat phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down the amino acid phenylalanine. Here, we delve into the market dynamics and financial trajectory of this crucial drug.

Market Size and Growth

The phenylketonuria treatment market, which includes sapropterin dihydrochloride, is experiencing significant growth. As of 2024, the market size for PKU treatments was valued at USD 0.84 billion and is projected to reach USD 1.86 billion by 2035, growing at a CAGR of around 7.5% between 2025 and 2035[1].

Regional Analysis

The North American market is the largest for PKU treatments, including sapropterin dihydrochloride, due to high awareness levels among both professionals and patients. Government initiatives and advanced healthcare infrastructure in this region contribute to its leadership. The Asia-Pacific region, however, is the fastest-growing market, driven by increasing healthcare infrastructure and growing disease awareness in countries like China, India, and Japan[1][4].

Treatment Type and Market Share

Sapropterin dihydrochloride is the most prominent medication in the PKU treatment market. It is widely used in both pediatric and adult populations to enhance phenylalanine metabolism in patients with tetrahydrobiopterin-responsive PKU. This medication holds a dominant position due to its approval and effectiveness, contributing significantly to the management of phenylalanine levels[1].

Financial Impact and Budget Analysis

The incorporation of sapropterin dihydrochloride into healthcare systems represents a significant budgetary impact. In Brazil, for example, the estimated incremental budget impact for including sapropterin dihydrochloride in the public healthcare system was around BRL 79 million (USD 21.7 million) in the first year and BRL 300 million (USD 82.1 million) over five years. The cost sensitivity analysis showed that the price of sapropterin dihydrochloride was the most sensitive variable in the model[2].

Cost-Utility Analysis

A cost-utility analysis comparing sapropterin dihydrochloride plus a phenylalanine-restricted diet with a diet alone showed significant incremental costs but also improved quality of life. The analysis indicated that sapropterin dihydrochloride plus diet was associated with additional quality-adjusted life years (QALYs) but at a high incremental cost per QALY, ranging from $274,862 to $308,664 depending on the response criteria used[5].

Generic Competition and Market Dynamics

The market for sapropterin dihydrochloride is also influenced by the emergence of generic versions. Dr. Reddy's Laboratories, for instance, launched a generic version of Kuvan in the United States in April 2021. This competition could impact the market share and pricing of the original drug, although BioMarin Pharmaceuticals, the original manufacturer, has reached settlements with generic manufacturers to delay their market entry[4].

New Market Entrants and Competitive Landscape

The approval of new drugs like Palynziq (pegvaliase) by BioMarin Pharmaceuticals adds to the competitive landscape. Palynziq is expected to achieve peak annual sales of over $500 million by 2029, which could offset potential declines in Kuvan sales due to generic competition. However, Palynziq's launch is expected to be slow due to its complex administration sequence and associated risks like anaphylaxis[3].

Government Funding and Support

Government funding and support for rare disease treatments are key growth drivers for the PKU treatment market. Initiatives to improve newborn screening programs and raise awareness about PKU in developing regions are crucial for expanding the market. These efforts not only increase the patient base but also foster a more supportive regulatory environment for new and existing treatments[1].

Challenges and Opportunities

Despite the growth, the market faces challenges such as the high cost of treatments. Financial difficulties due to the expensive nature of PKU treatments, including sapropterin dihydrochloride, are significant. However, opportunities lie in developing more affordable treatments and expanding market reach, especially in developing regions where awareness and healthcare infrastructure are improving[1][5].

Key Takeaways

• Market Growth: The PKU treatment market, driven by sapropterin dihydrochloride, is expected to grow at a CAGR of around 7.5% between 2025 and 2035.
• Regional Leadership: North America is the largest market, while the Asia-Pacific region is the fastest-growing.
• Financial Impact: Sapropterin dihydrochloride has a significant budgetary impact, with high incremental costs but improved quality of life.
• Generic Competition: The emergence of generic versions could impact market dynamics and pricing.
• New Entrants: New drugs like Palynziq are entering the market, adding to the competitive landscape.
• Government Support: Government initiatives and funding are crucial for market growth.

FAQs

1. What is the current market size for PKU treatments including sapropterin dihydrochloride? The market size for PKU treatments was valued at USD 0.84 billion in 2024[1].

2. What is the projected growth rate for the PKU treatment market? The market is expected to grow at a CAGR of around 7.5% between 2025 and 2035[1].

3. Which region is the largest market for PKU treatments? North America is the largest market for PKU treatments[1][4].

4. What is the impact of generic competition on sapropterin dihydrochloride? Generic versions, such as the one launched by Dr. Reddy's Laboratories, could reduce the market share and pricing of the original drug[4].

5. How does government funding influence the PKU treatment market? Government funding and support for rare disease treatments are key growth drivers, enabling better awareness and expanded treatment access[1].

Cited Sources:

1. Metatech Insights: Phenylketonuria Treatment Market Size and Forecast 2025-2035
2. Cambridge University Press: PP14 Budget Impact Of Sapropterin Dihydrochloride For Phenylketonuria
3. BioPharma Dive: BioMarin cleared to bring new PKU drug to market
4. Mordor Intelligence: Phenylketonuria Treatment Market - Share & Size
5. NCBI: Information on the Pharmacoeconomic Submission