Praxis Precision Medicines’ ulixacaltamide NDA for essential tremor has been accepted by the FDA, supported by positive Essential3 Phase 3 results showing significant improvement and maintained treatment effect.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
Praxis Precision Medicines has secured U.S. regulatory review for ulixacaltamide HCl, bringing the company closer to introducing a therapy specifically designed for adults with Essential Tremor. The U.S. Food and Drug Administration has accepted the New Drug Application and assigned a Prescription Drug User Fee Act target action date of January 29, 2027. The agency does not plan to convene an advisory committee meeting for the application.
The submission is supported by results from the Essential3 Phase 3 program (NCT06087276), which included two simultaneously enrolled pivotal studies in adults with essential tremor.
In the Essential3 program, patients treated with ulixacaltamide in the parallel-group Study 1 demonstrated a mean improvement of 4.3 points from baseline in Modified Activities of Daily Living-11 at Week 8, meeting the pre-specified primary endpoint (p<0.0001). All key secondary endpoints in Study 1, including rate of disease improvement over 12 weeks, Patient Global Impression of Change, and Clinical Global Impression–Severity, were also statistically significant (p<0.001). In Study 2, ulixacaltamide met its pre-specified primary endpoint, showing superior maintenance of effect versus placebo during the randomized-withdrawal phase (p=0.0369). The first key secondary endpoint in Study 2, rate of disease improvement during the randomized-withdrawal phase, also favored ulixacaltamide over placebo (p=0.0042), while other secondary endpoints were not statistically significant.
Ulixacaltamide was generally well tolerated, with a safety profile consistent with earlier studies and no drug-related serious adverse events reported. The therapy received Breakthrough Therapy Designation in December 2025.
Marcio Souza, president and chief executive officer said the FDA acceptance marks progress toward delivering a treatment designed specifically for patients with essential tremor, while the company prepares for potential commercialization during the review period.
Ulixacaltamide is a selective small-molecule inhibitor of T-type calcium channels. The therapy is designed to suppress abnormal neuronal burst firing within the cerebello-thalamo-cortical circuit, a pathway linked to tremor activity. The program represents the most advanced candidate within the company’s Cerebrum small-molecule platform.
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About the Writer
Chikkula Pavan Kumar, PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.