Generic Name: avapritinib
Drug Class: Targeted Therapy Medications
Company: Blueprint Medicines
Approval Status: Approved
Generic Version Available: No
Ayvakit is a kinase inhibitor approved for the treatment of inoperable or metastatic gastrointestinal stromal tumor (GIST) with specific PDGFRA mutations and for advanced systemic mastocytosis.
Ayvakit is a tyrosine kinase inhibitor that targets certain PDGFRA and KIT mutations that play a role in cancer cell growth. It is approved for GIST with PDGFRA exon 18 mutations, the most common of which is known as D842V.
The Phase I NAVIGATOR trial showed that among 43 GIST patients with PDGFRA exon 18 mutations, the overall response rate was 84%, with 7% having complete responses. Among the 38 people with PDGFRA D842V mutations, the overall and complete response rates rose to 89% and 8%, respectively. The Phase III VOYAGER trial is testing Ayvakit versus Stivarga (regorafenib) for GIST patients with extensive prior treatment. Ayvakit was first approved in 2020.
Ayvakit is a once-daily tablet that should be taken on an empty stomach.
Common side effects include swelling (edema), nausea, vomiting, abdominal pain, decreased appetite, diarrhea, constipation, fatigue, weakness, cognitive impairment, dizziness, rash, increased tears and hair color changes. Potential serious adverse events may include bleeding within the brain and central nervous system side effects. Ayvakit may cause fetal harm if used during pregnancy.
Last Reviewed: January 9, 2020