Avapritinib clinical trials
Avapritinib is named as an intervention in 8 current clinical trials in our China, Germany, UK, France, Italy, Netherlands, Belgium, Norway, Canada, USA, Spain, Sweden, Denmark and Switzerland registers. 6 are recruiting patients now. The most common phase is Phase 2. The sponsor with the most trials is Blueprint Medicines Corporation (3). The most-studied condition is Indolent Systemic Mastocytosis.
Where Avapritinib is approved
All regulators →Of the three regulators tracked here, the first to approve Avapritinib was the US, on 9 Jan 2020. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of mastocytosis (26 Oct 2018, positive)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Avapritinib patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Avapritinib, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 8 listed patents; the last listed expires 8 Mar 2042 (US 12060354).
- Latest patent on the drug substance itself: 10 Apr 2040 (US 11964980).
- Exclusivity ODE-434: Orphan drug exclusivity: treatment of adult patients with indolent systemic mastocytosis (ISM), until 22 May 2030.
- Exclusivity ODE-356: Orphan drug exclusivity: for the treatment of adult patients with advanced systemic mastocytosis (advsm). advsm includes patients with aggr…, until 16 Jun 2028.
- Exclusivity ODE-366: Orphan drug exclusivity: indicated for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth fac…, until 9 Jan 2027.
- AYVAKYT for treatment of gastrointestinal stromal tumours: until 25 Sep 2030. Union Register
- AYVAKYT for treatment of mastocytosis: until 25 Mar 2032. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Avapritinib
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Avapritinib works
All drug targets →- Stem cell growth factor receptor inhibitor InhibitorTarget: KIT KIT proto-oncogene, receptor tyrosine kinase✓ In ChEMBL and the Guide to Pharmacology
- Platelet-derived growth factor receptor alpha inhibitor InhibitorTarget: PDGFRA platelet derived growth factor receptor alphaChEMBL only
- Acute myeloid leukemiaPhase 2
- Core binding factor acute myeloid leukemiaPhase 2
- Metastatic malignant neoplasmPhase 2
- Systemic mastocytosisPhase 2
- Central nervous system cancerPhase 1/2
- Central nervous system neoplasmPhase 1/2
- Diffuse midline glioma, H3 K27-alteredPhase 1/2
- Hematopoietic and lymphoid cell neoplasmPhase 1
- Lymphoid neoplasmPhase 1
- Mast cell leukemiaPhase 1
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4204794, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 10368, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Avapritinib
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Avapritinib. 2 are current, and 1 of these recommend it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Avapritinib for treating advanced systemic mastocytosis NICE TA1012 | Recommended | 6 Nov 2024 | Current |
| Avapritinib for treating unresectable or metastatic gastrointestinal stromal tumours (terminated appraisal) NICE TA730 | Terminated (no company submission) | 29 Sep 2021 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Avapritinib
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 5 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: AyvakytEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Trials by status
Trials by phase
Clinical trials of Avapritinib
8 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Avapritinib across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
5 single-substance products containing Avapritinib on the HPRA medicines list.
- AYVAKYT 100 mg film-coated tabletsBlueprint Medicines (Netherlands) B.V. · EU/1/20/1473/001
- AYVAKYT 200 mg film-coated tabletsBlueprint Medicines (Netherlands) B.V. · EU/1/20/1473/002
- AYVAKYT 25 mg film-coated tabletsBlueprint Medicines (Netherlands) B.V. · EU/1/20/1473/004
- AYVAKYT 300 mg film-coated tabletsBlueprint Medicines (Netherlands) B.V. · EU/1/20/1473/003
- AYVAKYT 50 mg film-coated tabletsBlueprint Medicines (Netherlands) B.V. · EU/1/20/1473/005
Supply shortages
No entry for Avapritinib in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Avapritinib
9 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Blueprint Medicines Corporation | 3 |
| Children's Hospital of Soochow University | 2 |
| Ruijin Hospital | 1 |
| Institute of Hematology & Blood Diseases Hospital, China | 1 |
| Centre Leon Berard | 1 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Avapritinib trials
How many clinical trials are testing Avapritinib?
8 current trials in our China, Germany, UK, France, Italy, Netherlands, Belgium, Norway, Canada, USA, Spain, Sweden, Denmark and Switzerland registers name Avapritinib (also registered as BLU-285) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Avapritinib trials recruiting patients?
Yes. 6 of the 8 trials are recruiting: 3 with sites in China, 2 with sites in Germany, 1 with sites in UK, 1 with sites in France, 1 with sites in Italy, 1 with sites in Netherlands, 1 with sites in Belgium, 1 with sites in Norway and 1 with sites in Canada. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Avapritinib clinical trials?
Blueprint Medicines Corporation (3), Children's Hospital of Soochow University (2), Ruijin Hospital (1) and Institute of Hematology & Blood Diseases Hospital, China (1), among 5 lead sponsors in total. The number in brackets is how many trials each organisation leads.
Is Avapritinib licensed in Ireland?
Yes. The HPRA medicines list includes 5 single-substance products containing Avapritinib, for example AYVAKYT 100 mg film-coated tablets (Blueprint Medicines (Netherlands) B.V.). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Avapritinib shortage?
Not in the lists we track: Avapritinib has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Avapritinib works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Avapritinib works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Avapritinib (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.