Product Description
Avapritinib is used to treat a certain type of stomach, bowel, or esophagus cancer called gastrointestinal stromal tumor (GIST) that cannot be treated with surgery or that has spread to other parts of the body (metastatic), and that is caused by a certain platelet-derived growth factor receptor alpha (PDGFRA) gene mutation. This medicine is also used to treat advanced systemic mastocytosis (AdvSM), including aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). Avapritinib belongs to the group of medicines known as antineoplastics. It interferes with the growth of cancer cells, which are eventually destroyed. Since the growth of normal body cells may also be affected by sunitinib, other effects will also occur. Some of these may be serious and must be reported to your doctor. Other effects, like hair loss, may not be serious but may cause concern. Some effects may not occur for months or years after the medicine is used. (Sourced from: https://www.mayoclinic.org/drugs-supplements/avapritinib-oral-route/side-effects/drg-20484230?p=1)
Mechanisms of Action: KITl Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Austria | Belgium | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Italy | Latvia | Lithuania | Netherlands | Poland | Portugal | Romania | Slovakia | Spain | Sweden | Taiwan | United Kingdom | United States
Approved Indications: Oncology Unspecified
Known Adverse Events: Abdominal Pain | Dizziness | Pain Unspecified | Asthenia | Constipation | Diarrhea | Edema
Company: Royalty Pharma
Company Location:
Company CEO:
Additonal Commercial Interests: Blueprint Medicines
Clinical Description
Countries in Clinic: Austria, Belgium, Canada, China, Denmark, France, Germany, Italy, Korea, Netherlands, Norway, Poland, Spain, Sweden, Switzerland, United Kingdom, United States
Active Clinical Trial Count: 9
Highest Development Phases
Phase 3: Gastrointestinal Stromal Tumors
Phase 2: Glioma|Hypercalcemia|Mast-Cell Leukemia|Mastocytosis, Systemic|T-Cell Leukemia
Phase 1: Gastrointestinal Cancer|Myeloid Leukemia
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
---|---|---|---|---|---|
BLU-285-2203 | P2 |
Active, not recruiting |
Mastocytosis, Systemic |
2027-06-23 |
57% |
SM-AHN | P1 |
Recruiting |
Mastocytosis, Systemic |
2027-03-01 |
|
MegaMOST | P2 |
Recruiting |
Hypercalcemia |
2026-02-01 |
|
BLU-285-2202 | P2 |
Active, not recruiting |
T-Cell Leukemia|Mast-Cell Leukemia|Mastocytosis, Systemic |
2026-01-31 |
57% |