AYVAKIT Drug Patent Profile

When do Ayvakit patents expire, 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-two 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:	72
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
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

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
South Korea	20160062173	KIT에 관련된 장애를 치료하는데 유용한 조성물 (COMPOSITIONS USEFUL FOR TREATING DISORDERS RELATED TO KIT)
Serbia	64881
Portugal	3856341
Portugal	3856341

Supplementary Protection Certificates for AYVAKIT

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3057969	132021000000035	Italy		PRODUCT NAME: AVAPRITINIB E SUOI SALI FARMACEUTICAMENTE ACCETTABILI(AYVAKYT); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/20/1473, 20200925
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	122021000014	Germany		PRODUCT NAME: AVAPRITINIB UND PHARMAZEUTISCH VERTRAEGLICHE SALZE DAVON; REGISTRATION NO/DATE: EU/1/20/1473 20200924
3057969	2190008-9	Sweden		PRODUCT NAME: AVAPRITINIB OR PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; REG. NO/DATE: EU/1/20/1473 20200925

Market Dynamics and Financial Trajectory for AYVAKIT (Avapritinib)

Last updated: December 29, 2025

Summary

AYVAKIT (avapritinib) is a targeted therapy approved by the FDA for treating advanced systemic mastocytosis (SM) and gastrointestinal stromal tumors (GIST) harboring specific mutations. As a potent, selective tyrosine kinase inhibitor (TKI), AYVAKIT has carved a niche within precision oncology, addressing unmet clinical needs. This analysis delineates the current market landscape, regulatory trajectory, sales prospects, competitive environment, and key factors influencing AYVAKIT’s financial prospects, providing essential insights for stakeholders.

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What is AYVAKIT (Avapritinib)?

Attribute	Details
Generic Name	Avapritinib
Brand Name	AYVAKIT
Manufacturer	Blueprint Medicines
Approval Date	January 2020 (FDA), August 2022 (EMA) for specific indications
Indications	- Advanced systemic mastocytosis (SM)
- GIST with PDGFRA exon 18 mutations (e.g., D842V)
Mechanism	Selective tyrosine kinase inhibition, targeting KIT and PDGFRA mutations

Therapeutic niche: AYVAKIT boasts high selectivity for mutant kinases, leading to superior efficacy with fewer off-target effects, an attribute that underpins its market potential.

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How Do Market Dynamics Influence AYVAKIT's Commercial Trajectory?

1. Market Size and Patient Population

Indication	Estimated Patient Population	Source/Comment
Advanced systemic mastocytosis (SM)	~3,500 – 4,000 globally; primarily rare, with higher prevalence in adults	[1], [2]
Gastrointestinal stromal tumors (GIST) with PDGFRA D842V mutation	~1-3% of GIST cases; approximately 200-600 cases annually in the U.S.	[3], [4]

Note: Rarity limits total addressable market; however, high unmet need in these subpopulations fuels demand.

2. Competitive Landscape

Competitors	Key Features	Market Share	Notes
Other TKIs	Imatinib, sunitinib, regorafenib	Predominantly seen in wild-type GIST	Less effective for PDGFRA D842V mutant GIST
Emerging Agents	Ripretinib, avapritinib (AYVAKIT)	Growing	Targeted more specifically; AYVAKIT has an edge in D842V mutation
No direct competitors	For advanced SM	N/A	AYVAKIT currently the only approved agent for this indication

3. Regulatory Milestones & Approvals

Date	Milestone/Approval	Impact
Jan 2020	FDA approval for advanced SM	Initiated commercial sales in US
Aug 2022	EMA approval for SMA and GIST with PDGFRA exon 18 mutations	Expanding market access and revenue potential

4. Reimbursement & Pricing Strategies

Pricing (U.S.)	Details	Implications
Wholesale Acquisition Cost (WAC)	~$13,000 per month (approximate, varies by dosage)	Premium pricing reflects targeted therapy niche
Reimbursement	Favorable in US; coverage depends on indication	Critical for patient access; influences sales volume

5. Industry Policies & Market Access Dynamics

Policy Aspect	Details	Implication for AYVAKIT
Orphan Drug Designation	Granted in US and EU	Provides market exclusivity, tax incentives, and faster approval
Pricing and Access Policies	Increasing focus on fair pricing for rare diseases	May influence reimbursement negotiations
Market Exclusivity	7-year exclusivity in US (or longer in the EU), subject to patent life	Essential for maximizing profitability

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What Financial Trajectory Can Be Expected for AYVAKIT?

1. Revenue Projections (2023-2028)

Year	Estimated Global Sales ($ Millions)	Growth Rate	Factors Influencing Revenue
2023	150 – 200	—	Post-market penetration, expanding indications
2024	250 – 350	40–75%	Wider adoption, new label expansions, insurance coverage
2025	400 – 600	40–70%	Increasing mutation-specific indications, potential new approvals
2026	600 – 900	35–60%	Broader line extension, potential combination therapies
2027	900 – 1,200	25–50%	Market saturation in current indications, entry into additional markets

Note: These projections assume continued positive clinical data, expanded approvals, and favorable reimbursement.

2. Cost Structure & Profitability Outlook

• R&D Spending: Continues as pipeline activity persists; estimated at ~$50–$100 million annually.
• Manufacturing Costs: Marginal due to scalable synthesis; margins improve as sales grow.
• Pricing Power: High due to orphan status and niche efficacy, supporting gross margins of 80%+.

3. Risks & Challenges Impacting Financials

Risk Factor	Impact	Mitigation Strategy
Regulatory delays or denials	Revenue stagnation or reduction	Robust clinical trial program, communications
Emergence of resistance	Decreased efficacy over time	Combination therapies, next-generation inhibitors
Pricing & reimbursement pressures	Reduced profit margins	Demonstrate value, cost-effectiveness studies
Market expansion hurdles	Limited sales growth	Strategic partnerships, patient advocacy engagement

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How Does AYVAKIT Compare to Other Therapies?

Parameter	AYVAKIT (Avapritinib)	Rivals/Alternatives
Specificity for Mutations	High (PDGFRA D842V, KIT mutations)	Variable; less targeted
Approval Status	Fully approved for SM and GIST in select markets	Varies; some still in trials
Efficacy Metrics	ORR (Objective Response Rate): >70% in D842V GIST; significant symptom control in SM	Less effective for mutant GIST, variable in SM
Side Effect Profile	Manageable; most common: edema, fatigue, cognitive effects	Similar but less targeted TKIs may have broader side effects
Market Penetration	Early but increasing	Established in some markets but limited for rare mutations

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What Are the Critical Drivers for AYVAKIT's Future Growth?

Driver	Details	Implication
Expanded Label Indications	Beyond SM and D842V GIST, including other KIT mutations	Opens additional revenue streams
Combination Therapies	Synergistic effects with other agents	Enhances efficacy, overcomes resistance
Global Market Expansion	Entry into Asia-Pacific, Latin America, and EU markets	Significantly increases sales volume
Partnerships & Collaborations	Licensing deals, co-marketing, R&D collaborations	Accelerates development, broadens reach
Patient Advocacy & Awareness	Education for rare disease identification	Improves diagnosis rates and treatment uptake

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How Does the Future Outlook Look for AYVAKIT?

Scenario	Best-Case	Base-Case	Worst-Case
Market Penetration	Rapid expansion into multiple indications	Steady growth within current indications	Limited growth due to competition or regulatory setbacks
Sales Growth (2023-2028)	>200% cumulative increase	~150% growth	<50% growth or sales plateau
Pipeline Progress	New approvals and combinations	Moderate pipeline success	Delayed or failed pipeline developments

What Are the Key Takeaways for Stakeholders?

• Market Niche: AYVAKIT is positioned within a niche but high-need segment—rare mutations in GIST and advanced SM—favoring premium pricing and sustained demand.
• Growth Potential: Significant upside exists through label expansion, global penetration, and combination therapy development.
• Competitive Edge: Its mutation-specific mechanism grants it superior efficacy over broader-spectrum TKIs.
• Risks: Patent expiries, resistance development, reimbursement challenges, and emerging competitors could temper growth; strategic planning required.
• Financial Outlook: Projections indicate robust revenue growth over the next five years, contingent on regulatory success and market acceptance.

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Conclusion

AYVAKIT stands out as a targeted, mutation-specific therapy with promising clinical efficacy and a favorable regulatory environment. Its market trajectory hinges on strategic expansion into additional indications, global markets, and combination therapies. While it faces challenges common to orphan drugs, its high specificity and unmet need position it favorably for sustainable growth and profitability.

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FAQs

1. What are the primary factors driving AYVAKIT’s market growth?

Clinical efficacy in rare mutational subsets, FDA and EMA approvals, high unmet need, and favorable pricing strategies collectively fuel growth prospects.

2. How does AYVAKIT compare to other TKIs targeting GIST?

AYVAKIT exhibits higher selectivity for PDGFRA D842V mutations, leading to superior response rates in this subgroup, unlike broader spectrum TKIs like imatinib which are less effective for this mutation.

3. What regulatory hurdles could impact AYVAKIT’s financial trajectory?

Pending approvals for expanded indications, potential delays, or denials could limit market access; however, ongoing clinical trials aim to mitigate this risk.

4. How significant is the global market for AYVAKIT?

While the global patient population is small, high per-unit pricing, orphan drug incentives, and expanding indications promise meaningful revenue streams.

5. What strategies could enhance AYVAKIT’s market penetration?

Expanding indications, securing reimbursement, fostering global registrations, and developing combination regimens are key strategies.

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References

[1] Escribano L, et al. "Epidemiology and Management of Systemic Mastocytosis." Curr Hematol Malig Rep. 2020;15(1):1-13.
[2] Valent P, et al. "Advances in the Pathogenesis and Treatment of Systemic Mastocytosis." Hematol Oncol. 2022;40(4):385-395.
[3] Dematteo RP, et al. "Gastrointestinal stromal tumors." The Lancet. 2009;373(9664):1931-1944.
[4] Heinrich MC, et al. "Molecular Pathogenesis and Targeted Therapy of GIST." Nat Rev Clin Oncol. 2019;16(4):221-238.