Praxis Precision Medicines Secures FDA Breakthrough Therapy Designation for Ulixacaltamide

Praxis Precision Medicines, Inc. has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for ulixacaltamide hydrochloride for the treatment of essential tremor.

This designation is given to medicines for serious conditions where early clinical results show the potential for a clear improvement over existing treatments. 

It allows for faster development and closer collaboration with the FDA during the review process.

The decision is based on positive topline data from the Essential3 programme, which included two pivotal Phase 3 clinical trials in patients with essential tremor. 

These studies demonstrated that ulixacaltamide may offer meaningful benefits compared with currently available therapies.

Praxis has completed its pre-New Drug Application (NDA) discussions with the FDA and has aligned on the content required for submission. The company plans to file the NDA in early 2026.

Essential tremor is the most common movement disorder and affects around seven million people in the United States. 

It causes involuntary shaking, mainly in the hands and arms, and can significantly affect daily life. Current treatment options are limited and often poorly tolerated, leaving many patients without effective care.

Ulixacaltamide is a highly selective small-molecule inhibitor of T-type calcium channels designed to reduce abnormal nerve activity linked to tremor.