Praxis Precision Medicines Inc. announced on July 17, 2025, that the U.S. Food and Drug Administration has granted breakthrough therapy designation (BTD) for their drug relutrigine. This designation is specifically for pediatric use in treating patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The BTD facilitates expedited development and review processes for drugs that aim to treat serious conditions, where preliminary clinical evidence suggests substantial improvement over existing therapies. The designation was supported by positive results from the Phase 2 EMBOLD study and 11-month data from its open-label extension. Additionally, Praxis has commenced the EMERALD study to further evaluate relutrigine across all DEEs.
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