Avapritinib, supplied under the brand name Ayvakit, is a prescription medicine used to treat adults with PDGFRA GIST (gastrointestinal stromal tumour). This oral therapeutic drug targets specific mutations to prevent the growth and progression of PDGFRA GIST cancer cells. A mutation is an abnormal change to a gene, which may be responsible for causing disease.
What is Avapritinib for?
The medicine Avapritinib is for adults with bowel, stomach, or esophagus cancer called Gastrointestinal Stromal Tumor (GIST). Particularly, it’s for patients with a gastrointestinal stromal tumour that can’t be treated with the help of surgery or that has progressed to other body parts, and that has a mutation in the platelet-derived growth factor receptor alpha (PDGFRA) gene.
A healthcare provider will perform a test to make sure that the patient has this abnormal PDGFRA gene and that the medicine is right for them. It is not known if it is safe and promising for children.
Avapritinib- Clinical Trials and Efficacy:
Avapritinib was proven to be effective against GIST (gastrointestinal stromal tumour) with a PDGFRA exon 18 mutation. The medicine was evaluated in a clinical trial named NAVIGATOR. The trial enrolled a total of 43 individuals with PDGFRA exon 18 mutations, 38 of whom had the PDGFRA D842V mutation.
84% of 43 Individuals Responded:
33 individuals (77%) had their tumors shrink by at least 30% (partial response)
3 individuals (7%) had their tumors completely disappear (complete response)
In Addition:
- 6 individuals (14%) had no change in their tumor size (stable disease)
- 1 individual (2%) had their tumor grow (progressive disease)
Of the 38 individuals with a PDGFRA D842V mutation, 89% responded. Thirty-one individuals (82%) had a partial response (PR) and 3 individuals (8%) had a complete response (CR). It’s important to note that having a response does not mean the cancer has been cured. Individual results may vary.
References:
https://ayvakit.com/
https://gist.amegroups.org/article/view/65099/html
https://ir.blueprintmedicines.com/news-releases/news-release-details/blueprint-medicines-announces-updated-navigator-trial-results
