Praxis Precision Medicines Inc. experienced a notable surge in its stock price on Monday after the U.S. Food and Drug Administration (FDA) granted its drug candidate, ulixacaltamide, Breakthrough Therapy Designation (BTD) for the treatment of essential tremor (ET). This designation is integral in expediting both the development and regulatory assessment of therapies intended to treat serious medical conditions where preliminary evidence suggests the potential for substantial clinical benefit compared with existing treatments.
Essential tremor is recognized as the most widespread movement disorder, impacting an estimated seven million individuals across the United States alone. The condition is characterized primarily by involuntary, rhythmic shaking of the upper limbs and may also involve tremors affecting the head, vocal cords, or legs. Due to its prevalence and symptomatic burden, essential tremor represents a significant commercial opportunity, with a market size assessed in the multi-billion-dollar range.
Marcio Souza, president and chief executive officer of Praxis Precision Medicines, emphasized the progress made through recent interactions with the FDA. Mr. Souza highlighted that these discussions, combined with the granting of the Breakthrough Therapy Designation, position Praxis to accelerate the advancement of ulixacaltamide toward patients in need. He noted that the company is rigorously preparing for the submission of the new drug application (NDA) for ulixacaltamide, which is projected to occur in early 2026.
The FDA’s Breakthrough Therapy Designation is a regulatory mechanism designed to facilitate faster development and review processes for drugs that address serious conditions when preliminary clinical data suggests a meaningful improvement over existing therapeutic options. In the case of ulixacaltamide, this designation was awarded based on favorable topline data from Praxis’s Essential3 program. This program includes two pivotal Phase 3 clinical trials specifically evaluating the efficacy and safety of ulixacaltamide in patients with essential tremor.
In December of 2025, Praxis successfully completed its pre-NDA meeting with the FDA. This milestone included receiving written feedback and participating in an in-person meeting, which helped align the company with the FDA on the necessary content and structure of the upcoming NDA. The company’s proactive engagement with regulators reflects disciplined management and a strategic approach to navigating the development and approval pathway.
The positive market response to these developments has been clear. At the time of reporting on Monday, shares of Praxis Precision Medicines were trading up by 14.24%, reaching a price of $318.08. This price level signifies a new 52-week high for the stock, demonstrating strong investor enthusiasm following the BTD announcement.
Overall, the granting of Breakthrough Therapy Designation for ulixacaltamide elevates the visibility of Praxis’s clinical program in essential tremor. It also underscores the company’s potential to address a significant unmet medical need with a novel therapeutic option. The pathway forward involves rigorous regulatory submissions and further clinical scrutiny, but the progress to date marks a pivotal moment for both Praxis and patients affected by essential tremor.