Praxis Precision Medicines Announces the FDA Has Granted Breakthrough Therapy Designation for Ulixacaltamide HCl in Essential Tremor

Core Insights - The FDA has granted Breakthrough Therapy Designation (BTD) for ulixacaltamide, a selective small molecule inhibitor for treating essential tremor (ET) [1][3] - Praxis is preparing to submit the New Drug Application (NDA) for ulixacaltamide in early 2026 following positive interactions with the FDA [2][4] Industry Overview - Essential Tremor (ET) affects approximately seven million people in the U.S., representing a significant commercial opportunity [5] - Current treatment options for ET are limited, with propranolol being the only approved pharmacotherapy, which has poor tolerability and efficacy [5] - A substantial unmet need exists, as up to 77% of patients feel their ET is inadequately controlled, and around 50% are not receiving treatment [5] Company Overview - Praxis Precision Medicines focuses on developing therapies for CNS disorders using genetic insights [7] - The company has a diversified portfolio, including multiple clinical-stage product candidates targeting epilepsy and movement disorders [7] - Ulixacaltamide is the most advanced program within Praxis' Cerebrum™ small molecule platform, designed to block abnormal neuronal activity associated with tremors [6]