Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis
The European Medicines Agency (EMA) approved Blueprint Medicines’ label expansion of AYVAKYT® (avapritinib) to include monotherapy for the treatment of adult patients with rare blood disease advanced systemic mastocytosis (AdvSM), after at least one systemic therapy, in the EU, referencing real-world evidence (RWE) from a study co-investigated by an Analysis Group team led by Managing Principal Mei Sheng Duh. RWE generated by the observational retrospective cohort study involving chart review of real-world clinical data on best available therapies from six international centers of excellence provided an external control arm to help the EMA interpret single-arm Phase 1 EXPLORER and Phase 2 PATHFINDER trial findings.
Results from the study, “Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis,” which was coauthored by Dr. Duh, Vice President Priyanka Bobbili, Manager Aolin Wang, and Senior Analyst Selvam Sendhil, were published in Leukemia.
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Reiter A, Gotlib J, Álvarez-Twose I, Radia DH, Lübke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ