Praxis Precision Medicines (NASDAQ:PRAX) said the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to ulixacaltamide, a selective T-type calcium channel inhibitor being developed for the treatment of essential tremor, accelerating the regulatory path for the experimental therapy.
The company said the designation was based on positive topline data from the Essential3 program, which includes two pivotal Phase 3 trials evaluating ulixacaltamide in patients with essential tremor.
The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious conditions when preliminary clinical evidence suggests a substantial improvement over existing therapies.
“The granting of the Breakthrough Therapy Designation for ulixacaltamide further underscores its potential to address the substantial unmet need in patients with essential tremor,” President and Chief Executive Officer Marcio Souza said in a statement.
He added that recent positive interactions with the FDA, combined with the designation, position the company to advance the program more rapidly toward patients.
Praxis said it completed a pre–New Drug Application meeting with the FDA in December 2025, receiving both written feedback and holding an in-person discussion.
The company said it has aligned with the agency on the content of the NDA and expects to submit the application in early 2026.
Ulixacaltamide is a small-molecule inhibitor designed to modulate neuronal excitation-inhibition imbalance, a mechanism believed to underlie essential tremor, a common movement disorder for which treatment options remain limited.