> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Blueprint Medicines GIST Drug Approved
- URL: https://www.fdaweb.com/blueprint-medicines-gist-drug-approved/
- Published: 2020-01-09T12:00:00.000Z
- Updated: 2026-09-14T15:57:57.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5145814

FDA has approved a Blueprint Medicines NDA for Ayvakit (avapritinib) for treating adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) that have a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. Ayvakit is a kinase inhibitor, which blocks a type of enzyme called a kinase and helps keeps the cancer cells from growing, an FDA [release](https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-therapy-treat-rare-mutation-patients-gastrointestinal-stromal-tumors?ref=fdaweb.com) says. “Clinical trials showed a high response rate with almost 85% of patients experiencing tumor shrinkage with this targeted drug,” it says.

Approval is based on data from a clinical trial involving 43 patients with GIST harboring a PDGFRA exon 18 mutation, including 38 patients with PDGFRA D842V mutation, the agency says. “For patients harboring a PDGFRA exon 18 mutation, the overall response rate was 84%, with 7% having a complete response and 77% having a partial response,” it says. “For the subgroup of patients with PDGFRA D842V mutations, the overall response rate was 89%, with 8% having a complete response and 82% having a partial response. While the median duration of response was not reached, 61% of the responding patients with exon 18 mutations had a response lasting six months or longer (31% of patients with an ongoing response were followed for less than six months).”