Avapritinib granted FDA priority review of sNDA for the treatment of indolent SM

On January 23, 2022, the U.S. Food and Drug Administration (FDA) accepted a priority review to the supplemental new drug application for avapritinib, intended for the treatment of adults with indolent systemic mastocytosis (SM).¹ The FDA previously approved avapritinib for the treatment of adults with advanced SM in June 2021; however, there are currently no therapies available for the treatment of non-advanced SM.¹

This application was supported by results from the phase II PIONEER trial (NCT03731260) investigating the safety and efficacy of avapritinib versus placebo in patients with indolent SM.¹ Avapritinib produced a significant reduction in total symptom score and serum tryptase versus placebo (Table 1).¹

Table 1. Total symptom score and serum tryptase levels in patients treated with avapritinib and placebo*

In terms of safety, patients treated with avapritinib experienced lower rates of adverse events, including those deemed to be serious and cognitive (Table 2).¹

Table 2. Safety outcomes for patients treated with avapritinib and placebo*