Avapritinib

Oncology· 581 cited findings· 1 source document

Company: Blueprint Medicines B V

What the regulatory reviews say

Avapritinib is a drug for which a CHMP positive opinion was issued on 23 July 2020, following an application submission on 1 July 2019. It received orphan medicinal product designation on 17 July 2017. The recommended starting dose of avapritinib is 300 mg, with a reduced starting dose of 100 mg when administered with a moderate CYP3A inhibitor.

In studies, avapritinib demonstrated an Objective Response Rate (ORR) of 95% and a median Progression-Free Survival (PFS) of 24 months. The Duration of Response (DOR) was 22.1 months, and the 36-month Overall Survival (OS) rate was 70.6%. For patients with the PDGFRA D842V mutation, the median PFS was 3 to 5 months and the median OS was approximately 15 months. In a comparative study, the ORR in the avapritinib group was 42.9%, compared to 0% in the regorafenib group. The median PFS in the avapritinib group was not estimable, while for Sunitinib and regorafenib, the response rate was 5 to 7% with a median PFS of 5-6 months. Imatinib re-treatment showed an overall response rate of zero and a median PFS of 1.8 months, compared to a placebo median PFS of 0.9 months.

Regarding safety, 23% of patients experienced serious adverse reactions, with anaemia reported in 6% and pleural effusion in 1%. Treatment-related serious adverse events occurred in 20.1% of patients, including anaemia (4.3%), pleural effusion (1.7%), and acute kidney injury (1.3%). Photosensitivity reactions (Grade 1 or 2) were observed in 3% of patients. Dose interruptions or missed doses due to adverse events occurred in 63.7% of patients, and dose reductions due to adverse events occurred in 47.0% of patients.

Key figures — cited to the source page

Objective Response Rate (ORR)	95	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.105
median Progression-Free Survival (PFS)	24	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.105
Duration of Response (DOR)	22.1	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.105
Objective Response Rate (ORR) in avapritinib group	42.9	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.105
Objective Response Rate (ORR) in regorafenib group	0	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.105
Serious adverse reactions	23	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.112
CHMP positive opinion date	23 July 2020	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.9
Recommended starting dose of avapritinib	300	Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment, p.13

Source documents

Blueprint Medicines B V — Ayvakyt EMA EPAR public assessment report (EMEA/H/C/005208, 2020) (2020)

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Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.