Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment report (EMEA/H/C/005208, 2020)
Blueprint Medicines B V · 2020 · EMA EPAR public assessment report
Report summary
The EMA's 2020 assessment of Ayvakyt (avapritinib) by Blueprint Medicines B.V. led to conditional marketing authorization for unresectable/metastatic GIST with the PDGFRA D842V mutation, addressing an unmet medical need. Preclinical studies confirmed avapritinib's potent and selective inhibition of KIT and PDGFRα mutant enzymes, demonstrating efficacy in GIST models and acceptable toxicology. Clinical trials, particularly BLU-285-1101, showed outstanding efficacy in D842V-mutant GIST, with high objective response rates and durable responses. While safety data revealed substantial toxicities, including hematological, cardiovascular, and neurological adverse events, these were deemed manageable with risk minimization. Post-authorization measures include submitting results from ongoing trials and an observational safety study to confirm long-term safety and efficacy.
Key findings — cited to the page
| Patients with AdvSM experiencing Grade 2 or Grade 3 cognitive effects | 12 % | · p.116 |
| Patients with GIST with decreased leukocyte counts shifted from Grade <=2 to Grade >=3 | 4.3 % | · p.121 |
| Patients with GIST experiencing Grade >=3 respiratory AEs | 6.8 % | · p.120 |
| Patients with GIST experiencing Grade >=3 GI AEs | 22.6 % | · p.119 |
| Patients with GIST with decreased hemoglobin values shifted from Grade <=2 to Grade >=3 | 24.4 % | · p.121 |
| Patients with GIST with decreased neutrophil counts shifted from Grade <=2 to Grade >=3 | 5.6 % | · p.121 |
| Photosensitivity reactions (Grade 1 or 2) | 3 % | · p.50 |
| Patients with GIST experiencing Grade >=3 General Disorders and Administration Site Conditions SOC AEs | 17.5 % | · p.120 |
| Patients with GIST experiencing Grade 2 or Grade 3 cognitive effects | 13 % | · p.116 |
| median time to a Grade 3 or 4 AE for 1st quartile of Cave | 10 months | · p.78 |
| median time to a Grade 3 or 4 AE for 4th quartile of Cave | 2 to 3 months | · p.78 |
| patients with decreased platelet counts shifted from Grade <=2 at baseline to Grade >=3 on study | 1 | · p.122 |
Source: Blueprint Medicines B V ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →