Drugs / Avapritinib
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (2) | US (FDA) | AYVAKIT | — | 2021-06-16 – 2023-05-22 | fda.gov ↗ |
| Approved · conditional | EU (EMA) | Ayvakyt | — | 2020-09-24 | europa.eu ↗ |
| Approved | US (FDA) | AYVAKIT | — | 2020-01-09 | fda.gov ↗ |
Trials 22
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-02-28 | Blueprint Medicines Corporation | Results Blueprint Medicines, a Sanofi Company, Announces Four-Year PIONEER Data Showing Sustained Benefit and Long-Term Safety of AYVAKIT® (avapritinib) in Indolent Systemic Mastocytosis blueprintmedicines.com ↗
Key presentations include four-year PIONEER clinical study results highlighting sustained symptom control and quality of life improvement in AYVAKIT-treated patients, and real-world data showing meaningful symptom benefit in patients receiving the therapy in a community practice setting. |
| 2025-12-05 | Blueprint Medicines Corporation | Results Blueprint Medicines, a Sanofi Company, Highlights Long-Term Clinical Benefit of AYVAKIT® (avapritinib) Across Indolent and Advanced Systemic Mastocytosis at 2025 ASH Annual Meeting blueprintmedicines.com ↗
Blueprint Medicines, a Sanofi company, today announced updated data reinforcing the clinical efficacy and safety of long-term AYVAKIT® (avapritinib) use across the spectrum of systemic mastocytosis (SM), including indolent and advanced SM. |
| 2025-06-12 | Blueprint Medicines Corporation | Results Blueprint Medicines Announces Data Reinforcing Sustained Clinical Efficacy and Well-Tolerated Safety Profile of Long-Term AYVAKIT®/AYVAKYT® (avapritinib) Treatment at 2025 EHA and EAACI Congresses blueprintmedicines.com ↗
AYVAKIT has shown transformative clinical outcomes for patients across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM. |
| 2025-02-28 | Blueprint Medicines Corporation | Results Blueprint Medicines Highlights Leading Portfolio of Mast Cell-Targeted Therapies at 2025 AAAAI / WAO Joint Congress blueprintmedicines.com ↗
Updated data show AYVAKIT® (avapritinib) was safe and effective for long-term treatment of ISM, with sustained symptom and quality-of-life benefit and no new safety signals with multiple years of therapy |
| 2024-12-07 | Blueprint Medicines Corporation | Results Blueprint Medicines' Continued Leadership in Redefining the Standard of Care in Systemic Mastocytosis Highlighted at 2024 ASH Annual Meeting blueprintmedicines.com ↗
ASH data reinforce survival benefits of front-line AYVAKIT® (avapritinib) use in patients with advanced SM |
| 2024-05-30 | Blueprint Medicines Corporation | Results Blueprint Medicines Announces Data Presentations at EAACI and EHA Annual Meetings Highlighting Sustained Clinical Benefits of AYVAKIT®/AYVAKYT® (avapritinib) blueprintmedicines.com ↗
With a median follow-up of more than two years, AYVAKIT showed durable efficacy and a favorable safety profile in patients with ISM. |
| 2024-02-23 | Blueprint Medicines Corporation | Results Blueprint Medicines Highlights AYVAKIT® (avapritinib) Long-Term Efficacy and Safety Data and Advances in Mast Cell Disease Research at 2024 AAAAI Annual Meeting blueprintmedicines.com ↗
Long-term data from the PIONEER trial show that AYVAKIT led to robust improvements across all symptom domains (skin, gastrointestinal, neurocognitive) at 24 weeks, with sustained benefits through 48 weeks. |
| 2023-05-22 | Blueprint Medicines Corporation | Regulatory FDA Approves AYVAKIT® (avapritinib) as the First and Only Treatment for Indolent Systemic Mastocytosis blueprintmedicines.com ↗
Blueprint Medicines Corporation (Nasdaq: BPMC) today announced that the U.S. Food and Drug Administration (FDA) has approved AYVAKIT ® (avapritinib) for the treatment of adults with indolent systemic mastocytosis (ISM). |
All press releases naming this drug 11 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 26 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Avapritinib | “Avapritinib is a type 1 kinase inhibitor designed to potently and selectively inhibit oncogenic KIT/PDGFRA mutants by targeting the kinase active conformation.” PMID 36477870 ↗ Feb 2023 |
| Known as | 70C366 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794 ↗ |
| Known as | Ayvakit | ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794 ↗ |
| Known as | Ayvakyt | ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794 ↗ |
| Known as | BLU-285 | “This phase 1 study ( NCT02561988 ) evaluated avapritinib (BLU-285), a selective KIT D816V inhibitor, in patients with AdvSM.” PMID 34873347 ↗ Dec 20211“This record comprises the currently available Expanded Access Programs (EAP) for avapritinib (BLU-285):” NCT04714086 ↗ |
| Known as | C-366 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794 ↗ |
| Known as | X-720776 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794 ↗ |
| Action | Inhibit | “Avapritinib selectively and potently inhibits KIT- and PDGFRA-mutant kinases.” PMID 34343033 ↗ Aug 2021 |
| Modality | Small molecule | “a small molecular targeted agent (Avapritinib)” NCT07028073 ↗ |
| Route | Oral | “administered orally (PO)” NCT02561988 ↗ |
| Target | KIT | “Avapritinib selectively and potently inhibits KIT- and PDGFRA-mutant kinases.” PMID 34343033 ↗ Aug 2021 |
| Target | PDGFRA | “Avapritinib selectively and potently inhibits KIT- and PDGFRA-mutant kinases.” PMID 34343033 ↗ Aug 2021 |