“Praxis Precision Medicines’ ulixacaltamide HCl earns FDA BTD for essential tremor based on Essential3 Phase 3 data (NCT06087276). NDA expected early 2026 new hope beyond propranolol”
Written By: Pharmacally Medical News Desk
Praxis Precision Medicines announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for Ulixacaltamide HCl, an investigational treatment for essential tremor (ET). The designation, based on positive topline results from the Essential3 Phase 3 clinical program, is designed to accelerate the development and regulatory review of promising therapies for serious conditions with unmet needs.
The FDA’s Breakthrough Therapy Designation is reserved for treatments that show early clinical evidence of substantial improvement over existing therapies for serious diseases. BTD does not mean the drug is approved, but it allows for more intensive FDA guidance and a potentially faster review timeline as the company prepares a New Drug Application (NDA). Praxis has said it expects to submit the NDA for Ulixacaltamide in early 2026 following recent alignment with FDA on submission content.
Ulixacaltamide (PRAX-944) is a differentiated, highly selective T-type calcium channel inhibitor designed to reduce abnormal neuronal activity believed to contribute to essential tremor symptoms. This mechanism targets the cerebello-thalamo-cortical circuit involved in tremor generation.
The Breakthrough designation was supported by top-line outcomes from the Essential3 Phase 3 program (NCT06087276), which consisted of two pivotal studies. In the first study, patients receiving Ulixacaltamide showed statistically significant improvements in the modified Activities of Daily Living 11-item scale (mADL11), a measure of daily function impacted by tremor. The second study demonstrated sustained treatment effects during a randomized withdrawal phase. Overall, the data indicated meaningful benefit in tremor control and daily functioning compared with placebo.
Essential tremor is one of the most common movement disorders, affecting millions of people worldwide and particularly impacting daily activities such as eating, writing, and dressing. Current FDA-approved options are limited. Beta-blockers like propranolol are widely used but many patients find them only modestly effective and difficult to tolerate. The new designation for Ulixacaltamide highlights both its potential and the gap in effective treatment choices.
With Breakthrough Therapy Designation secured, Praxis is preparing to submit an NDA to the FDA in early 2026. The company has already completed a pre-NDA meeting with FDA, received written agency feedback, and gained alignment on key elements of the submission. If approved, Ulixacaltamide could become an important new option for people with essential tremor, expanding the therapeutic landscape beyond existing treatments.
Experts in movement disorders have noted that a therapy showing consistent functional improvements could significantly benefit people whose tremors interfere with basic tasks. As a medication specifically developed for essential tremor rather than extrapolated from other uses, Ulixacaltamide represents a potentially meaningful advance in care.
Essential tremor is characterized by involuntary, rhythmic shaking, usually in the hands and arms, making precise movements difficult. The condition can worsen over time and affect quality of life. Although not life-threatening, the functional limitations can be significant. Current standard treatments only partly address symptoms for many patients.
The FDA’s Breakthrough Therapy Designation for Ulixacaltamide HCl marks a significant regulatory milestone for Praxis Precision Medicines and raises expectations for a new targeted treatment for essential tremor. With positive Phase 3 data and a clear path toward NDA submission, this program is among the most anticipated in movement disorder therapeutics
References
Praxis Precision Medicines Announces the FDA Has Granted Breakthrough Therapy Designation for Ulixacaltamide HCl in Essential Tremor, 29 December 2025, https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-fda-has-granted
Essential 3 – Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET), ClinicalTrials.gov ID NCT06087276, https://clinicaltrials.gov/study/NCT06087276
Essential tremor Diagnosis and Treatment, Mayo Clinic, https://www.mayoclinic.org/diseases-conditions/essential-tremor/diagnosis-treatment/drc-20350539