Avapritinib - Generic Drug Details

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

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

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

One supplier is listed for this compound.

Summary for avapritinib

International Patents:	70
US Patents:	7
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	41
Clinical Trials:	13
Patent Applications:	116
What excipients (inactive ingredients) are in avapritinib?	avapritinib excipients list
DailyMed Link:	avapritinib at DailyMed

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

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 avapritinib

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

Sponsor	Phase
The First Affiliated Hospital of Soochow University	Phase 2
Theseus Pharmaceuticals	Phase 1/Phase 2
Blueprint Medicines Corporation	Phase 4

See all avapritinib clinical trials

Pharmacology for avapritinib

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

Anatomical Therapeutic Chemical (ATC) Classes for avapritinib

L01EX	Other protein kinase inhibitors
L01E	PROTEIN KINASE INHIBITORS
L01	ANTINEOPLASTIC AGENTS
L	Antineoplastic and immunomodulating agents

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	212608-001	Jan 9, 2020		RX	Yes	No
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			Y	Y
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-002	Jan 9, 2020		RX	Yes	No
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-004	Jun 16, 2021		RX	Yes	No			Y	Y
Blueprint Medicines	AYVAKIT	avapritinib	TABLET;ORAL	212608-004	Jun 16, 2021		RX	Yes	No			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/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 avapritinib

Country	Patent Number	Title	Estimated Expiration
Brazil	112016008541
Japan	2019048878
Mexico	2016004927
Mexico	365614
Philippines	12016500611
Portugal	3409674

Supplementary Protection Certificates for avapritinib

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3057969	10/2021	Austria		PRODUCT NAME: AVAPRITINIB UND PHARMAZEUTISCH ANNEHMBARE SALZE DAVON; REGISTRATION NO/DATE: EU/1/20/1473 (MITTEILUNG) 20200925
3057969	122021000014	Germany		PRODUCT NAME: AVAPRITINIB UND PHARMAZEUTISCH VERTRAEGLICHE SALZE DAVON; REGISTRATION NO/DATE: EU/1/20/1473 20200924
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; REG. NO/DATE: 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	CA 2021 00008	Denmark		PRODUCT NAME: AVAPRITINIB OG FARMACEUTISK ACCEPTABLE SALTE DERAF; REG. NO/DATE: EU/1/20/1473 20200925

Market Dynamics and Financial Trajectory for Avapritinib

Introduction

Avapritinib, marketed as AYVAKIT or AYVAKYT, is a groundbreaking drug approved by the FDA for the treatment of unresectable or metastatic gastrointestinal stromal tumor (GIST) with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including the D842V mutation. Here, we delve into the market dynamics and financial trajectory of this significant therapeutic advancement.

FDA Approval and Clinical Efficacy

Avapritinib received FDA approval on January 9, 2020, marking it as the first therapy specifically approved for patients with GIST harboring a PDGFRA exon 18 mutation. The drug demonstrated an overall response rate (ORR) of 84% in clinical trials, with 7% complete responses and 77% partial responses for patients with PDGFRA exon 18 mutations, and an ORR of 89% for those with the D842V mutation[4].

Market Penetration and Adoption

The approval and subsequent market introduction of avapritinib have been met with significant market penetration. The drug has shown robust performance, particularly in the treatment of Indolent Systemic Mastocytosis (ISM), in addition to its primary indication for GIST.

• Revenue Growth: In 2023, avapritinib generated $204.2 million in net product revenues, with a notable $71.0 million in the fourth quarter alone. This performance has led to an optimistic outlook for 2024, with projected revenues ranging from $435 million to $450 million, representing over 80% year-over-year growth[3][5].

• Market Share: The introduction of avapritinib has led to a significant shift in market share. For unresectable or metastatic GIST with a PDGFRA exon 18 mutation, avapritinib's market share is projected to increase to 45%, 60%, and 80% in years 1, 2, and 3, respectively, at the expense of other treatments like imatinib, sunitinib, regorafenib, and nilotinib[1].

Financial Performance

The financial performance of avapritinib has been stellar, driven by its strong market adoption and the chronic nature of the conditions it treats.

• Quarterly Revenues: In the second quarter of 2024, avapritinib achieved $114.1 million in net product revenues, representing a year-over-year growth of more than 185%[2].

• Full-Year Projections: The company has raised its full-year revenue guidance for avapritinib to $435 million to $450 million, reflecting continued strong execution in the global launch for ISM and other indications[2][5].

• Cost of Sales: The cost of sales has remained relatively flat, primarily due to the increased sales of lower-cost dosages of avapritinib. This has helped in maintaining a favorable cost structure[5].

Economic Impact on Health Plans

The economic evaluation of avapritinib's impact on health plans indicates a minimal budget impact.

• Budget Impact Model: A 3-year budget impact model showed that the introduction of avapritinib would result in a total increase in costs of $115,604, or $0.010 per member per month (PMPM), for a hypothetical 1-million member health plan. This includes an offset of $3,607 in postprogression costs avoided or delayed[1].

• Testing and Monitoring Costs: The model also accounted for increased PDGFRA testing rates and minimal incremental testing costs, which were estimated at $453 in year 3[1].

Sensitivity Analysis

To assess the robustness of the financial projections, a one-way deterministic sensitivity analysis was conducted.

• Key Parameters: The analysis involved increasing and decreasing key parameters such as drug price, treatment duration, variant rate, molecular testing rate, postprogression pharmacy costs, and treated market share for avapritinib by a factor of 20%. This analysis was performed using Microsoft Excel[1].

Long-Term Revenue Drivers

The long-term financial trajectory of avapritinib is driven by several key factors:

• Treatment Adherence and Persistence: The chronic nature of ISM and other treated conditions means that patients remain on therapy for extended periods, contributing to stable and growing revenue streams[3].

• Positive Clinical Data: Continued positive long-term data on the safety and efficacy of avapritinib can lead to increased trust among healthcare providers, resulting in higher adoption rates and longer treatment durations[3].

Key Takeaways

• Strong Market Adoption: Avapritinib has shown significant market penetration and adoption, particularly in the treatment of ISM and GIST.
• Robust Financial Performance: The drug has generated substantial revenues, with projected growth exceeding 80% year-over-year.
• Minimal Budget Impact: The introduction of avapritinib has a minimal impact on health plan budgets.
• Long-Term Revenue Potential: The chronic nature of treated conditions and positive clinical data ensure stable and growing revenue streams.

FAQs

What is avapritinib used for?

Avapritinib is used for the treatment of unresectable or metastatic gastrointestinal stromal tumor (GIST) with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including the D842V mutation, and also for Indolent Systemic Mastocytosis (ISM)[4].

How effective is avapritinib in clinical trials?

Avapritinib demonstrated an overall response rate (ORR) of 84% in clinical trials for patients with PDGFRA exon 18 mutations, and an ORR of 89% for those with the D842V mutation[4].

What is the projected revenue for avapritinib in 2024?

The projected revenue for avapritinib in 2024 is approximately $435 million to $450 million, representing over 80% year-over-year growth[2][5].

What is the impact of avapritinib on health plan budgets?

The introduction of avapritinib results in a minimal budget impact, with a total increase in costs of $115,604, or $0.010 per member per month (PMPM), for a hypothetical 1-million member health plan[1].

What are the key drivers of long-term revenue for avapritinib?

The key drivers include treatment adherence and persistence due to the chronic nature of treated conditions, and positive long-term clinical data that can increase adoption rates and treatment durations[3].

Sources

1. Financial Implications of Avapritinib for Unresectable GIST. JAMA Network Open, 2020.
2. Blueprint Medicines Reports Second Quarter 2024 Results and Raises AYVAKIT Full Year Revenue Guidance. Blueprint Medicines, 2024.
3. Blueprint Medicines Reports Fourth Quarter and Full Year 2023. Stock Titan, 2024.
4. FDA Approves Avapritinib for GIST with a Rare Mutation. FDA, 2020.
5. Blueprint Medicines Reports Strong First Quarter 2024 Results and Raises AYVAKIT Full Year Revenue Guidance. PR Newswire, 2024.