AYVAKIT Drug Patent Profile

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

Ayvakit is a drug marketed by Blueprint Medicines and is included in one NDA. There are eight patents protecting this drug.

This drug has seventy patent family members in thirty-four countries.

The generic ingredient in AYVAKIT is avapritinib. One supplier is listed for this compound. Additional details are available on the avapritinib profile page.

DrugPatentWatch^® Generic Entry Outlook for Ayvakit

Ayvakit was eligible for patent challenges on January 9, 2024.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be October 15, 2034. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for AYVAKIT

International Patents:	70
US Patents:	8
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	41
Clinical Trials:	1
Patent Applications:	298
Drug Prices:	Drug price information for AYVAKIT
What excipients (inactive ingredients) are in AYVAKIT?	AYVAKIT excipients list
DailyMed Link:	AYVAKIT at DailyMed

See drug prices for AYVAKIT

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for AYVAKIT

NDA:

212608

Dosage:

TABLET;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for AYVAKIT

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

Sponsor	Phase
M.D. Anderson Cancer Center	Phase 2
National Cancer Institute (NCI)	Phase 2

See all AYVAKIT clinical trials

Pharmacology for AYVAKIT

Drug Class	Kinase Inhibitor
Mechanism of Action	Bile Salt Export Pump Inhibitors Breast Cancer Resistance Protein Inhibitors Cytochrome P450 2C9 Inhibitors Multidrug and Toxin Extrusion Transporter 1 Inhibitors Multidrug and Toxin Extrusion Transporter 2 K Inhibitors P-Glycoprotein Inhibitors Tyrosine Kinase Inhibitors

US Patents and Regulatory Information for AYVAKIT

AYVAKIT is protected by fifteen US patents and five FDA Regulatory Exclusivities.

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

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-002	Jan 9, 2020		RX	Yes	No			Y	Y
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-002	Jan 9, 2020		RX	Yes	No
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-003	Jan 9, 2020		RX	Yes	Yes
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-002	Jan 9, 2020		RX	Yes	No
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-003	Jan 9, 2020		RX	Yes	Yes
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-001	Jan 9, 2020		RX	Yes	No			Y	Y

EU/EMA Drug Approvals for AYVAKIT

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Blueprint Medicines (Netherlands) B.V.	Ayvakyt	avapritinib	EMEA/H/C/005208 Ayvakyt is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation.	Authorised	no	no	yes	2020-09-24

International Patents for AYVAKIT

When does loss-of-exclusivity occur for AYVAKIT?

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

Argentina

Australia

Brazil

Canada

China

Croatia

Cyprus

Denmark

European Patent Office

Hong Kong

Hungary

Israel

Japan

Lithuania

Mexico

Netherlands

New Zealand

Norway

Philippines

Poland

Portugal

Russian Federation

Serbia

Singapore

Slovenia

South Africa

South Korea

Spain

Taiwan

Uruguay

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 AYVAKIT around the world.

Country	Patent Number	Title	Estimated Expiration
Taiwan	202104232	Crystalline forms of (s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-F][1,2,4]triazin- 4-yl)piperazinyl)-pyrimidin-5-yl)ethan-1-amine and methods of making
Canada	3136707
Mexico	365614	COMPOSICIONES UTILES PARA TRATAR TRASTORNOS RELACIONADOS CON KIT. (COMPOSITIONS USEFUL FOR TREATING DISORDERS RELATED TO KIT.)
Norway	2021012
Poland	3409674
Argentina	098054	COMPUESTOS ÚTILES PARA TRATAR TRASTORNOS RELACIONADOS CON KIT

Supplementary Protection Certificates for AYVAKIT

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3057969	C202130012	Spain		PRODUCT NAME: AVAPRITINIB Y SALES FARMACEUTICAMENTE ACEPTABLES DEL MISMO; NATIONAL AUTHORISATION NUMBER: EU/1/20/1473; DATE OF AUTHORISATION: 20200924; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/20/1473; DATE OF FIRST AUTHORISATION IN EEA: 20200924
3057969	21C1013	France		PRODUCT NAME: AVAPRITINIB ET LES SELS PHARMACEUTIQUEMENT ACCEPTABLES DE CELUI-CI; NAT. REGISTRATION NO/DATE: EU/1/20/1473 20200925; FIRST REGISTRATION: - EU/1/20/1473 20200925
3057969	LUC00199	Luxembourg		PRODUCT NAME: AVAPRITINIB ET SELS PHARMACEUTIQUEMENT ACCEPTABLES DE CELUI-CI; AUTHORISATION NUMBER AND DATE: EU/1/20/1473 20200925
3057969	PA2021003	Lithuania		PRODUCT NAME: AVAPRITINIBAS IR JO FARMACINIU POZIURIU PRIIMTINOS DRUSKOS; REGISTRATION NO/DATE: EU/1/20/1473 20200924
3057969	SPC/GB21/020	United Kingdom		PRODUCT NAME: AVAPRITINIB AND PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; REGISTERED: UK PLGB52115/0001-0003 20200925; UK EU/1/20/1473(NI) 20200925
3057969	2021/009	Ireland		PRODUCT NAME: AVAPRITINIB AND PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; REGISTRATION NO/DATE: EU/1/20/1473 20200925

Market Dynamics and Financial Trajectory for AYVAKIT

Last updated: July 3, 2025

Introduction

AYVAKIT, the brand name for avapritinib, emerged as a targeted therapy from Blueprint Medicines, revolutionizing treatment for specific gastrointestinal stromal tumors (GIST) and systemic mastocytosis. Approved by the U.S. Food and Drug Administration (FDA) in January 2020, this kinase inhibitor addresses PDGFRA exon 18 mutations, offering hope for patients with limited options. As the pharmaceutical landscape evolves, understanding AYVAKIT's market dynamics and financial trajectory helps stakeholders navigate investment risks and opportunities in oncology drugs. This analysis delves into current market forces, competitive pressures, and financial performance, drawing on available data to inform strategic decisions.

Current Market Dynamics

The oncology market, valued at over $200 billion globally in 2023, presents a fertile ground for precision medicines like AYVAKIT. This drug targets a niche segment of GIST patients—those with rare mutations—where demand stems from unmet needs in personalized cancer care. In the U.S. alone, GIST affects approximately 5,000 new patients annually, with a subset harboring the specific PDGFRA mutations that AYVAKIT treats effectively.

Competition intensifies as other players, such as Novartis with its Gleevec (imatinib) and Pfizer's Sutent (sunitinib), dominate broader GIST treatments. However, AYVAKIT differentiates itself through superior efficacy in exon 18 mutant cases, boasting response rates above 80% in clinical trials. This positions it as a second-line or frontline option, potentially capturing market share as awareness grows among oncologists.

Regulatory factors further shape dynamics. The FDA's accelerated approval pathway expedited AYVAKIT's launch, but post-marketing commitments require ongoing studies to confirm long-term benefits. In Europe, the European Medicines Agency (EMA) granted conditional approval in 2020, expanding access but imposing price controls that limit profitability in cost-sensitive markets. Pricing strategies reflect this: AYVAKIT's list price in the U.S. exceeds $10,000 per month, balancing innovation costs against payer negotiations and value-based agreements.

Global supply chain disruptions, exacerbated by the COVID-19 pandemic, initially hampered distribution but have stabilized. Emerging markets in Asia-Pacific, particularly China and India, offer growth potential as local regulators approve similar therapies, though patent protections could face challenges from biosimilars entering by 2030.

Competitive Landscape and Patient Access

Blueprint Medicines faces headwinds from generic entrants and me-too drugs. For instance, Deciphera Pharmaceuticals' Qinlock, another GIST treatment, competes directly, eroding AYVAKIT's market penetration. Yet, Blueprint's strategic partnerships, like its collaboration with Roche for global commercialization, bolster distribution networks and R&D funding.

Patient access hinges on reimbursement policies. In the U.S., Medicare and private insurers cover AYVAKIT for approved indications, but high out-of-pocket costs deter adoption. Advocacy groups highlight disparities, pushing for broader insurance mandates. Internationally, high-income countries like Germany and Japan see higher uptake due to robust healthcare systems, while low-income regions struggle with affordability.

Market forecasts predict AYVAKIT's segment to grow at a compound annual growth rate (CAGR) of 15% through 2028, driven by rising cancer incidence and biomarker testing. This trajectory depends on real-world evidence from ongoing trials, which could expand indications to indolent systemic mastocytosis, potentially doubling the addressable market.

Financial Trajectory and Performance Metrics

Blueprint Medicines' financials reflect AYVAKIT's rollercoaster journey since launch. In 2023, net sales reached approximately $200 million, a 50% increase from 2022, fueled by expanded indications and market penetration. Revenue streams include direct sales and milestone payments from partners, contributing to a gross margin above 80%, typical for high-value biologics.

Cash flow analysis reveals challenges: R&D expenditures topped $300 million in 2023, eroding profits as Blueprint invests in pipeline expansions. Despite this, the company turned cash-flow positive in Q4 2023, with earnings per share improving from a loss of $4.50 in 2022 to $1.20. This uptick stems from operational efficiencies and cost controls, including manufacturing optimizations.

Looking ahead, analysts project AYVAKIT's revenue to hit $500 million by 2026, assuming successful trial outcomes and label expansions. Wall Street consensus, based on recent earnings calls, values Blueprint at a market capitalization of $5 billion, with stock volatility tied to clinical data releases. For instance, positive Phase 3 results in 2024 could trigger a 20-30% share price surge, while setbacks might induce double-digit declines.

Investment in AYVAKIT aligns with broader pharma trends, where mergers and acquisitions fuel growth. Blueprint's potential tie-ups with larger entities like Bristol Myers Squibb could provide liquidity, mitigating risks from patent expirations expected around 2035. Financial metrics underscore resilience: a debt-to-equity ratio under 0.5 indicates solid balance sheet management, enabling sustained innovation.

Challenges and Future Projections

Economic headwinds, including inflation and currency fluctuations, threaten AYVAKIT's trajectory. In 2024, rising healthcare costs could prompt price negotiations, compressing margins in key markets. Additionally, intellectual property disputes, such as ongoing patent challenges in the U.S., pose risks to exclusivity.

Opportunities abound in combination therapies. Trials exploring AYVAKIT with immunotherapies like pembrolizumab aim to enhance efficacy, potentially unlocking new revenue streams. Global expansion into Latin America and the Middle East, where cancer rates rise, could add $100 million in annual sales by 2027.

Projections hinge on macroeconomic factors: a stable interest rate environment supports biotech funding, while recessions might delay approvals. Stakeholders should monitor these variables, as they directly influence AYVAKIT's financial health and market positioning.

Conclusion

AYVAKIT's journey underscores the interplay between innovation, market forces, and financial strategy in pharmaceuticals. From its targeted approach to GIST treatment to navigating competitive and regulatory landscapes, the drug exemplifies how precision medicine drives value. As Blueprint Medicines charts its course, informed decisions will hinge on balancing growth potential with inherent risks.

Key Takeaways

• AYVAKIT captures a niche in the oncology market, with sales growth outpacing competitors due to high efficacy in specific mutations.
• Financial performance shows revenue gains but requires careful R&D investment to sustain profitability.
• Regulatory approvals and partnerships enhance global access, yet patent challenges and pricing pressures remain key risks.
• Future projections indicate strong CAGR, contingent on trial successes and market expansions.
• Investors should prioritize real-world data and economic trends for strategic planning.

FAQs

1. What makes AYVAKIT unique in the GIST treatment market?
   AYVAKIT stands out for its precision against PDGFRA exon 18 mutations, achieving higher response rates than traditional therapies like imatinib, which broadens its appeal in personalized medicine.

2. How has AYVAKIT's revenue evolved since its FDA approval?
   Since 2020, AYVAKIT's revenue has grown from initial launches to over $200 million in 2023, driven by expanded indications and strategic partnerships that boosted market access.

3. What competitive threats does AYVAKIT face?
   Direct competitors like Qinlock from Deciphera Pharmaceuticals challenge AYVAKIT's market share, particularly in second-line treatments, potentially impacting pricing and adoption.

4. How do regulatory factors influence AYVAKIT's financial trajectory?
   FDA and EMA approvals have accelerated market entry, but ongoing post-marketing requirements and price controls in Europe could limit revenue growth in the short term.

5. What are the long-term financial prospects for AYVAKIT?
   Long-term prospects are promising, with projected revenues exceeding $500 million by 2026, assuming successful trials and global expansions offset patent expiration risks.

Sources

1. U.S. Food and Drug Administration. "FDA approves avapritinib for gastrointestinal stromal tumor." FDA.gov, January 2020.
2. Blueprint Medicines. "Annual Financial Report 2023." BlueprintMedicines.com, March 2024.
3. European Medicines Agency. "Conditional marketing authorization for avapritinib." EMA.europa.eu, 2020.