AYVAKIT Drug Patent Profile

When do Ayvakit patents expire, and when can generic versions of Ayvakit launch?

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-one 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.

There have been two patent litigation cases involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

Indicators of Generic Entry

Summary for AYVAKIT

International Patents:	71
US Patents:	8
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	41
Clinical Trials:	1
Patent Applications:	555
Drug Prices:	Drug price information for AYVAKIT
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases 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
National Cancer Institute (NCI)	Phase 2
M.D. Anderson Cancer Center	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

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/005208Ayvakyt 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

San Marino

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
Croatia	P20181388
Mexico	2016004927
Hungary	E064283

Supplementary Protection Certificates for AYVAKIT

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3057969	2021C/509	Belgium		PRODUCT NAME: AVAPRITINIB ET SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; AUTHORISATION NUMBER AND DATE: EU/1/20/1473 20200925
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	PA2021003	Lithuania		PRODUCT NAME: AVAPRITINIBAS IR JO FARMACINIU POZIURIU PRIIMTINOS DRUSKOS; REGISTRATION NO/DATE: EU/1/20/1473 20200924

Market Dynamics and Financial Trajectory for AYVAKIT

Last updated: July 27, 2025

Introduction

AYVAKIT (avapritinib) has established itself as a pivotal therapeutic agent in the oncology sector, specifically targeting mutated forms of the c-KIT and PDGFRA kinases. Approved by the U.S. Food and Drug Administration (FDA) in 2020 for the treatment of systemic mastocytosis and gastrointestinal stromal tumors (GIST) with specific mutations, AYVAKIT’s market trajectory reflects evolving oncology treatment paradigms, competitive pressures, and pipeline developments. Analyzing the current market dynamics and projecting AYVAKIT’s financial outlook involves dissecting its clinical positioning, regulatory landscape, competitive environment, and target patient populations.

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Market Overview and Clinical Positioning

AYVAKIT’s approved indications position it distinctly within niche but high-value oncology segments. For systemic mastocytosis, a rare mast cell disease with limited treatment options, AYVAKIT offers targeted therapy addressing the underlying genetic mutations. Its rapid adoption is fortified by the unmet need in this rare disease. Conversely, for GIST harboring PDGFRA D842V mutations, a subset representing approximately 5-10% of GIST cases, AYVAKIT provides a targeted alternative after resistance or intolerance to standard therapies.

Market size considerations depend heavily on the accruing prevalence of these disease subtypes. Systemic mastocytosis affects approximately 1 in 10,000 to 20,000 individuals globally, translating into a small but significant patient pool with high unmet needs. GIST, albeit more prevalent overall (annual incidence estimated at 4,000-6,000 cases in the U.S.), with the D842V mutation constituting a minority, limits the broader market but offers a lucrative niche.

Market Drivers

1. Unmet Medical Need: The scarcity of effective treatments for rare mast cell disorders and specific GIST mutations underpins AYVAKIT’s value proposition. Clinical data demonstrating high response rates bolster physician adoption.

2. Regulatory Approvals and Label Expansion: Continued regulatory support, coupled with real-world evidence, could endorse indications beyond initial approvals, expanding potential markets.

3. Specialty Physician Adoption: Disease specialists, including hematologists and oncologists, form the core prescriber base. Education and awareness campaigns influence uptake rates.

4. Tide of Clinical Research: Ongoing trials assessing AYVAKIT in other indications (e.g., systemic eosinophilic disorders) could eventually diversify revenue streams.

Competitive Landscape

AYVAKIT operates in a highly competitive realm characterized by:

• Existing TKIs: Imatinib, sunitinib, and regorafenib, though less tailored, are used off-label or after failure of other therapies in GIST. Their broader risk profiles, however, afford AYVAKIT a positioning advantage in mutation-specific subtypes.

• Emerging Agents: Other novel agents targeting similar mutations are in early clinical phases, threatening AYVAKIT’s market share if superior efficacy emerges.

• Therapeutic Shift to Precision Medicine: Increasing adoption of genetic testing enhances AYVAKIT’s utility, reinforcing its position as a mutation-specific drug.

Pricing and Reimbursement Factors

Cost considerations are central, with AYVAKIT being priced approximately at a premium level reflective of specialized oncology therapies (~$15,000 per month, estimates vary). Payer coverage hinges on demonstrated clinical benefit, cost-effectiveness, and alignment with treatment guidelines. Favorable reimbursement will anchor revenue streams.

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Financial Trajectory and Revenue Projections

The financial outlook for AYVAKIT hinges on multiple factors:

• Initial Revenue Generation (2021–2022): Post-launch, early adoption in US and select international markets generated modest revenues, primarily from systemic mastocytosis, with global sales reaching approximately $100 million in 2022 according to analyst estimates.

• Market Penetration and Growth (2023–2027): As clinical familiarity grows and additional indications are pursued, revenues are forecasted to Accelerate. A compounded annual growth rate (CAGR) of 25-30% appears plausible, driven by increasing patient diagnosis, expanded indications, and geographical expansion, with projected revenues reaching $400-$600 million by 2027.

• Pipeline and Lifecycle Management: The potential approval of AYVAKIT in broader GIST indications, as well as other mast cell diseases, can substantially elevate financial growth trajectories. Conversely, patent expirations and biosimilar threats in broader markets could dampen future profitability, though limited immediate competition in niches sustains margins.

• Pricing and Cost Dynamics: As manufacturing complexity evolves, costs may rise slightly; however, larger volumes and optimized supply chains are expected to mitigate margin erosions. The sustained premium pricing supports healthy profit margins.

Market Risks and Challenges

• Regulatory Hurdles: Further approvals in other indications are not guaranteed, and delays may impede revenue acceleration.

• Pricing Pressures: Payers in multiple markets may impose stringent reimbursement criteria, constraining sales.

• Competitive Innovation: The advent of next-generation inhibitors or combination therapies could reduce AYVAKIT’s market share.

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Emerging Trends Influencing Market and Financial Trajectory

1. Genetic Testing and Precision Medicine: Increasing adoption of comprehensive genomic profiling enhances identification of eligible patients, expanding the addressable market.

2. Regulatory Shifts: Potential fast-track designations and expanded label claims could accelerate market penetration.

3. Market Expansion Strategies: Collaborations with healthcare providers and policy shifts toward personalized therapies could amplify sales.

4. Global Market Penetration: Emerging markets, including Europe, Asia, and Latin America, present growth opportunities, albeit with regulatory and reimbursement hurdles.

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Key Takeaways

• AYVAKIT’s market prospects are buoyed by its targeted mechanism of action in niche, high-need patient populations, ensuring a steady revenue base.
• Continued clinical development, regulatory support, and strategic market expansion are vital to sustaining revenue growth through 2027.
• Competition from existing TKIs and emerging therapies necessitates ongoing differentiation through clinical efficacy, safety profile, and pricing strategies.
• Government and payer acceptance, coupled with evolving healthcare policies favoring precision medicine, bolster AYVAKIT’s financial trajectory.
• Long-term success relies on pipeline diversification, indication expansion, and global commercialization.

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FAQs

1. What are the primary indications for AYVAKIT?
   AYVAKIT is approved for treating systemic mastocytosis and gastrointestinal stromal tumors (GIST) harboring PDGFRA D842V mutations.

2. How does AYVAKIT compare to competitors?
   Its mutation-specific targeting offers advantages over broader-spectrum TKIs, enabling higher efficacy in selected patient subsets with favorable safety profiles.

3. What are the main growth drivers for AYVAKIT’s market?
   Increasing awareness, expanded indications, genetic testing adoption, and geographic expansion propel growth.

4. What risks could impede AYVAKIT’s financial outlook?
   Regulatory delays, pricing pressures, and emerging competitive therapies pose significant challenges.

5. Is AYVAKIT being investigated for other indications?
   Yes, ongoing clinical trials are evaluating its efficacy in other mast cell-related disorders and rare cancers, potentially broadening its market.

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References

1. FDA Approvals for AYVAKIT
2. Global market analysis reports (e.g., IQVIA, EvaluatePharma)
3. Published clinical trial data (e.g., NCT numbers related to AYVAKIT trials)
4. Industry financial disclosures and analyst estimates