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Praxis Precision Medicines Announces the FDA Has Granted Breakthrough Therapy Designation for Ulixacaltamide HCl in Essential Tremor
The Breakthrough Therapy Designation was granted based on the positive topline results from the Essential3 Phase 3 program in essential tremor Praxis remains
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"The Breakthrough Therapy Designation was granted based on the positive topline results from the Essential3 Phase 3 program in essential tremor Praxis remains on track to submit ulixacaltamide NDA in early 2026 based on recently completed pre-NDA meeting with the FDA BOSTON, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for ulixacaltamide, a differentiated and highly selective small molecule inhibitor of T-type calcium channels, for the treatment of patients with essential tremor (ET). “The granting of the Breakthrough Therapy Designation for ulixacaltamide, based on the Essential3 program, further underscores its potential to address the substantial unmet need in patients with ET. We recently completed a series of positive interactions with the FDA, that, together with this BTD, are enabling us to advance this promising treatment faster to patients. We are diligently preparing for the filing of the ulixacaltamide NDA, which we expect in early 2026,” said Marcio Souza, president and chief executive officer. The BTD enables expedited development and regulatory review for drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. The BTD for ulixacaltamide was based on the positive topline data from the Essential3 program, consisting of two pivotal Phase 3 studies of ulixacaltamide in ET. In December 2025, Praxis announced the successful completion of its pre-NDA meeting with the FDA, including the receipt of written feedback and an in-person meeting. The Company has aligned with the FDA on the content of the NDA and expects to submit the ulixacaltamide NDA in early 2026. About Essential Tremor (ET)Essential Tremor is the most common movement disorder, affecting roughly seven million people in the United States alone, representing a multi-billion dollar commercial opportunity. ET is characterized by involuntary rhythmic m...