avapritinib - Profile

What are the generic drug sources for avapritinib and what is the scope of patent protection?

Avapritinib is the generic ingredient in one branded drug marketed by Blueprint Medicines and is included in one NDA. There are eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Avapritinib has seventy-three patent family members in thirty-four countries.

Summary for avapritinib

International Patents:	73
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for avapritinib

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

Constraining patent/regulatory exclusivity:

9,200,002

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.

US Patents and Regulatory Information for avapritinib

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	212608-004	Jun 16, 2021		RX	Yes	No	11,999,744
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-004	Jun 16, 2021		RX	Yes	No	9,200,002		Y	Y
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-004	Jun 16, 2021		RX	Yes	No	12,252,494
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-004	Jun 16, 2021		RX	Yes	No	9,944,651		Y	Y
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-004	Jun 16, 2021		RX	Yes	No	9,994,575		Y	Y

EU/EMA Drug Approvals for avapritinib

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 avapritinib

Country	Patent Number	Title	Estimated Expiration
Lithuania	3057969
Taiwan	I683814
Japan	7728178
Canada	3296895
Netherlands	301094

Supplementary Protection Certificates for avapritinib

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3057969	LUC00199	Luxembourg		PRODUCT NAME: AVAPRITINIB ET SELS PHARMACEUTIQUEMENT ACCEPTABLES DE CELUI-CI; AUTHORISATION NUMBER AND DATE: EU/1/20/1473 20200925
3057969	132021000000035	Italy		PRODUCT NAME: AVAPRITINIB E SUOI SALI FARMACEUTICAMENTE ACCETTABILI(AYVAKYT); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/20/1473, 20200925
3057969	2190008-9	Sweden		PRODUCT NAME: AVAPRITINIB OR PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; EG EU/1/20/1473, 2020-09-25; PRV HAR I BESLUT DEN 16 JULI 2025 RAETTAT SKYDDSTIDEN I FOELJANDE TILLAEGGSSKYDD I ENLIGHET MED PMD:S BESLUT PMAE 7804/24: 1990003-4 2190008-9
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	PA2021003	Lithuania		PRODUCT NAME: AVAPRITINIBAS IR JO FARMACINIU POZIURIU PRIIMTINOS DRUSKOS; REGISTRATION NO/DATE: EU/1/20/1473 20200924

Avapritinib: Investment Scenario, Market Dynamics, and Financial Trajectory

Last updated: February 3, 2026

Summary

Avapritinib (brand name Ayvakit) is a targeted tyrosine kinase inhibitor developed by Blueprint Medicines, primarily approved for rare gastrointestinal tumors and specific mutations. Its market potential hinges on its specificity, clinical efficacy, regulatory approvals, and the landscape of targeted oncology therapies. This report evaluates the investment landscape, market dynamics, and projected financial trajectory of avapritinib, considering recent approvals, market size, competitive environment, and growth drivers.

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1. Overview of Avapritinib’s Medical and Regulatory Profile

Parameter	Details
Indications	- Advanced gastrointestinal stromal tumors (GIST) with PDGFRA exon 18 mutations (FDA, 2020)
	- Systemic mastocytosis (SM), including aggressive systemic mastocytosis (ASM) and systemic mastocytosis with associated hematologic neoplasm (SM-AHN) (FDA, 2020)
Approval Date	May 9, 2020 (FDA)
Mechanism of Action	Selective inhibitor of PDGFRA exon 18 mutations and KIT mutations involved in GIST, and KIT D816V mutation in SM.
Pricing (2023)	Approximate list price: $15,000 per month per patient (varies by indication and payer negotiations).

2. Market Size and Growth Potential

2.1 Current Market and Penetration

Segment	Estimated Global Market Size (USD, 2023)	Notes
GIST with PDGFRA exon 18 mutations	~$500 million	Rare mutation subset; ~3-5% of GIST cases (~7,000 annual cases).
Systemic mastocytosis	~$1 billion	Estimated global cases ~3,000-4,000 patients; orphan indication.
Total Addressable Market (TAM)	~$1.5 billion	Potential, with access expansion, especially in developed markets.

2.2 Market Drivers

• Advances in molecular diagnostics increase detection of PDGFRA exon 18 mutations and KIT D816V mutations.
• Increasing approval of avapritinib for broader indications (e.g., pediatric GIST, additional systemic mastocytosis subtypes).
• Growing awareness and diagnosis rates of rare diseases.

2.3 Market Limitations

• Orphan drug designation restricts pricing pressure but limits market size.
• Competition from other TKIs like imatinib, ripretinib, and newer agents.
• Uncertain off-label use and reimbursement landscape.

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3. Competitive Environment

Key Competitors	Indication Focus	Market Share (2023)	Strengths	Weaknesses
Imatinib (Gleevec)	GIST, CML	~60% in GIST (older therapies)	Well-established, broad indications, proven efficacy	Resistance in PDGFRA exon 18 mutations
Ripretinib (Qapzola)	GIST (advanced)	Emerging (~10-15%)	Approved for GIST after imatinib	Limited data in rare mutations
Avapritinib (Ayvakit)	GIST, SM	Growing (~15-20%)	Highly selective, effective in select mutations	Competition with emerging therapies
Other emerging TKIs	GIST, SM	Niche players	Target specific mutations	Less established, limited clinical data

4. Regulatory and Reimbursement Landscape

• FDA fast-track and orphan drug designations accelerate approval and market access.
• Payer reimbursement typically favorable given the orphan status; however, high pricing can limit access.
• Ongoing negotiations with payers to expand coverage.

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5. Financial Trajectory and Investment Outlook

5.1 Revenue Projections (2023–2030)

Year	Estimated Market Penetration	Annual Revenue (USD Millions)	Basis
2023	10% of TAM	~$150 million	Initial uptake; early adoption in specialized centers
2024-2025	20–30% of TAM	~$300–$450 million	Expanded indications and geographic expansion
2026-2027	40–50% of TAM	~$600–$750 million	Broader indication approvals, increased diagnostic testing
2028+	60–80% of TAM	~$900 million–$1.2 billion	Market saturation, off-label use, and best-practice standards

5.2 Key Drivers of Revenue Growth

• Expansion of approved indications: Additional tumor types and pediatric use.
• Diagnostic advancements: Increased detection rates.
• Pricing strategy: Maintaining premium pricing via orphan benefits.
• Market penetration: Geographic expansion (e.g., Asia, Europe).

5.3 Risks to Financial Trajectory

Risk Factor	Impact
Regulatory delays or rejections	Halt or slowdown in revenue growth
Competitive erosion	Loss of market share to emerging therapies
Pricing and reimbursement pressures	Profit margins compression
Off-label use and generics (post-patent)	Market dilution after exclusivity expires

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6. Policy and Industry Trends Impacting Avapritinib

Trend	Implication for Avapritinib
Personalized medicine	Enhances demand for mutation-specific TKIs
Orphan drug policy evolution	Maintains incentives, but increasing scrutiny over pricing and access
Digital health and diagnostics	Supports early detection and appropriate targeted therapy deployment
Market consolidation	Potential partnerships or M&A activity to enhance market reach

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7. Comparative Analysis with Key Oncology Drugs

Parameter	Avapritinib	Imatinib	Ripretinib	Other TKIs
Type	Selective TKI	Multi-kinase TKI	Switch-control kinase inhibitor	Selective TKIs
Indications	Rare GIST, SM	CML, GIST	GIST (post-imatinib)	Various oncogene-driven cancers
Market Size (USD)	~$1.5 billion (2023)	~$10 billion	~$1 billion	Variable
Time to Market Entry	2020	2001	2020	Varies
Pricing	$15,000/month	$10,000–$15,000/month	Similar but varies	Varies

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8. Conclusion and Investment Outlook

Avapritinib offers compelling growth prospects within niche but expanding markets driven by molecular diagnostics and orphan designation incentives. Its financial trajectory depends on continued approval expansion, market access, and competitive positioning. Risks include emerging therapies and reimbursement challenges, but its specificity and recent acceptance position it as a valuable asset in the targeted oncology therapeutic space.

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Key Takeaways

• Market Size & Growth: Estimated global market of ~$1.5 billion, with potential to reach over $2 billion by 2030.
• Revenue Potential: Projected revenues of $600M–$1.2B within the next five years, contingent on indication expansion and uptake.
• Competitive Landscape: Dominated by older TKIs with limited efficacy in certain mutations; avapritinib holds a niche advantage due to mutation specificity.
• Regulatory & Policy Environment: Orphan status offers favorable pricing and incentives but demands careful navigation of reimbursement.
• Investment Considerations: Risk factors include market competition, regulatory hurdles, and pricing pressures; opportunity centers on its targeted niche and ongoing clinical developments.

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FAQs

1. What are the primary drivers for avapritinib's market growth?

Advances in molecular diagnostics leading to increased detection, approval expansion for broader indications, and high unmet needs in rare tumors. The orphan drug designation also incentivizes market entry and premium pricing.

2. How does avapritinib compare to other TKIs in terms of efficacy?

Avapritinib demonstrates high specificity and efficacy in PDGFRA exon 18-mutant GIST and KIT D816V-mutant systemic mastocytosis, outperforming broader TKIs like imatinib in these mutation subsets.

3. What are the main hurdles for avapritinib’s future growth?

Emerging competitors, regulatory delays, reimbursement limitations, and potential off-label use impacting pricing strategies pose significant challenges.

4. Which regions are most promising for avapritinib’s expansion?

North America and Europe remain primary markets, with Asia-Pacific offering growth opportunities as diagnostic capabilities improve.

5. What is the expected patent and exclusivity period for avapritinib?

Patent protection extends until approximately 2030–2035, with orphan drug exclusivity potentially adding 7 years of market exclusivity post-approval.

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Sources

[1] FDA Label for Ayvakit (Avapritinib), 2020.
[2] Blueprint Medicines Corporate Reports, 2023.
[3] Global Data Estimates, 2023.
[4] Market Access and Pricing Policies, 2023.
[5] Competitive Oncology Therapeutics Market Reports, 2023.