Avapritinib

Generic Name: Avapritinib

Brand Names: Ayvakit, Ayvakyt

Drug Type: Small Molecule

Chemical Formula: C_₂₆H_₂₇FN_₁₀

CAS Number: 1703793-34-3

Unique Ingredient Identifier: 513P80B4YJ

Background: Avapritinib, or BLU-285, is a selective tyrosine kinase inhibitor of KIT and platelet derived growth factor receptor alpha indicated for the treatment of unresectable, metastatic gastrointestinal stromal tumors and advanced systemic mastocytosis. It is one of the first medications available for the treatment of multidrug resistant cancers. Avapritinib shares a similar mechanism with ripretinib.

Avapritinib was granted FDA approval on 9 January 2020 and EMA approval on 24 September 2020.

Indication: Avapritinib is indicated for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

It is also used to treat adult patients with advanced systemic mastocytosis (AdvSM). AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia. However, it is not recommended for the treatment of patients with AdvSM with platelet counts of less than 50 X 10 L.

Associated Conditions: Advanced Systemic Mastocytosis (AdvSM), Aggressive Systemic Mastocytosis, Indolent Systemic Mastocytosis, Mast Cell Leukemia (MCL), Metastatic Gastrointestinal Stromal Tumor (GIST), Systemic Mastocytosis With an Associated Hematological Neoplasm, Unresectable Gastrointestinal stromal tumor

Clinical Trials

Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors

Phase 2

Recruiting

Conditions: Anatomic Stage IV Breast Cancer AJCC v8
Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8
Clinical Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8
Clinical Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8
Locally Advanced Malignant Solid Neoplasm
Locally Advanced Melanoma
Locally Advanced Primary Malignant Central Nervous System Neoplasm
Locally Advanced Sarcoma
Metastatic Malignant Solid Neoplasm
Metastatic Melanoma

Interventions: Drug: Avapritinib

First Posted Date: 2021-02-25

Last Posted Date: 2025-02-17

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 50

Registration Number: NCT04771520

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States

Expanded Access Program for Avapritinib

Conditions: Solid Tumors

First Posted Date: 2021-01-19

Last Posted Date: 2024-10-24

Lead Sponsor: Blueprint Medicines Corporation

Registration Number: NCT04714086

A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/characteristics in Advanced / Metastatic Tumors.

Phase 2

Recruiting

Conditions: Malignant Solid Tumor

Interventions: Drug: Cabozantinib
Drug: Trametinib
Drug: HDM201
Drug: Regorafenib
Drug: Alectinib
Drug: Avapritinib
Drug: Ribociclib
Drug: Dabrafenib

First Posted Date: 2019-10-04

Last Posted Date: 2024-12-12

Lead Sponsor: Centre Leon Berard

Target Recruit Count: 455

Registration Number: NCT04116541

Locations: 🇫🇷
Institut Bergonié, Bordeaux, France
🇫🇷
Centre Léon Bérard, Lyon, France
🇫🇷
Institut Paoli Calmettes, Marseille, France

and more 5 locations

Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic GIST

Conditions: GIST

First Posted Date: 2019-03-05

Last Posted Date: 2020-07-27

Lead Sponsor: Blueprint Medicines Corporation

Registration Number: NCT03862885

(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

Phase 2

Active, not recruiting

Conditions: Indolent Systemic Mastocytosis

Interventions: Drug: Placebo
Drug: Avapritinib

First Posted Date: 2018-11-06

Last Posted Date: 2024-08-07

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 251

Registration Number: NCT03731260

Locations: 🇺🇸
Winship Cancer Institute, Emory University, Atlanta, Georgia, United States
🇺🇸
Dana Farber Cancer Institute, Boston, Massachusetts, United States
🇩🇪
University Clinic Hamburg Eppendorf, University Cancer Center Hamburg (UCCH), Hamburg, Germany

and more 46 locations

(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis

Phase 2

Completed

Conditions: Systemic Mastocytosis With an Associated Hematologic Neoplasm
Mast Cell Leukemia
Aggressive Systemic Mastocytosis
Advanced Systemic Mastocytosis

Interventions: Drug: Avapritinib

First Posted Date: 2018-07-09

Last Posted Date: 2025-04-30

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 107

Registration Number: NCT03580655

Locations: 🇨🇦
St. Michael's Hospital, Toronto, Ontario, Canada
🇺🇸
Dana Farber Cancer Institute, Boston, Massachusetts, United States
🇺🇸
University of Texas, MD Anderson Cancer Center, Houston, Texas, United States

and more 29 locations

(VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST

Phase 3

Completed

Conditions: GIST

Interventions: Drug: avapritinib
Drug: regorafenib

First Posted Date: 2018-03-14

Last Posted Date: 2022-10-06

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 476

Registration Number: NCT03465722

Locations: 🇺🇸
Fox Chase Cancer Center, Philadelphia, Pennsylvania, United States
🇺🇸
Northwestern Medicine, Chicago, Illinois, United States
🇺🇸
The University of Chicago Medical Center, Chicago, Illinois, United States

and more 109 locations

(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies

Phase 1

Completed

Conditions: Relapsed or Refractory Myeloid Malignancies
Systemic Mastocytosis-associated Hematologic Non-mast Cell Disease
Mast Cell Leukemia
Aggressive Systemic Mastocytosis

Interventions: Drug: Avapritinib

First Posted Date: 2015-09-29

Last Posted Date: 2023-03-17

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 86

Registration Number: NCT02561988

Locations: 🇺🇸
Huntsman Cancer Institute, Salt Lake City, Utah, United States
🇬🇧
Beatson West of Scotland Cancer Centre, Glasgow, United Kingdom
🇺🇸
Stanford Cancer Institute, Stanford, California, United States

and more 8 locations

(NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

Phase 1

Completed

Conditions: Gastrointestinal Stromal Tumors (GIST)
Other Relapsed or Refractory Solid Tumors

Interventions: Drug: Avapritinib

First Posted Date: 2015-07-27

Last Posted Date: 2022-06-21

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 250

Registration Number: NCT02508532

Locations: 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States
🇺🇸
Oregon Health and Science University, Portland, Oregon, United States
🇩🇪
University of Duisburg-Essen, Essen, Germany

and more 16 locations

Drug Development Updates

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