The Committee for Medicinal Products for Human Use adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product Ayvakyt (avapritinib), from Blueprint Medicines, intended for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. Ayvakyt will be available as film-coated tablets (100 mg, 200 mg and 300 mg).
Avapritinib, a protein kinase inhibitor, is designed to potently and selectively inhibit oncogenic KIT and PDGFRA mutants by targeting the active conformation of the kinase. The benefits with Ayvakyt are its ability to provide durable responses in patients with GIST harbouring the PDGFRA D842V mutation. The most common side effects (in at least 20% of patients) are nausea, fatigue, anaemia, periorbital oedema, face oedema, hyperbilirubinaemia, diarrhoea, vomiting, oedema peripheral, lacrimation increased, decreased appetite and memory impairment.