Praxis Precision Medicines Wins FDA Breakthrough Therapy Designation For Ulixacaltamide, A Selective T-Type Calcium Channel Inhibitor For Essential Tremor

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company focused on using genetic insights to develop therapies for central nervous system disorders linked to imbalances in neuronal excitation and inhibition, announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation for ulixacaltamide. Ulixacaltamide is a highly selective small-molecule inhibitor of T-type calcium channels and is being developed for the treatment of essential tremor, a common and often disabling movement disorder.

According to Marcio Souza, president and chief executive officer of Praxis, receiving Breakthrough Therapy Designation highlights ulixacaltamide’s potential to meet an important unmet medical need for patients living with essential tremor. He noted that the company recently completed a series of constructive interactions with the FDA, which, along with this designation, will help accelerate the development timeline. Praxis is now preparing the New Drug Application for ulixacaltamide and expects to submit it in early 2026.

Breakthrough Therapy Designation is intended to speed the development and review of medicines targeting serious conditions when early clinical data suggest substantial improvement over existing therapies. The designation for ulixacaltamide is based on positive topline results from the Essential3 program, which includes two pivotal Phase 3 clinical trials evaluating the drug in patients with essential tremor.

In December 2025, Praxis reported that it had successfully completed its pre-NDA meeting with the FDA. This meeting included both written feedback and an in-person discussion, and the company confirmed alignment with the FDA on the overall content and structure of the upcoming NDA. Praxis remains on track to file the ulixacaltamide NDA in early 2026.