Potential Essential Tremor Tx Receives FDA Breakthrough Therapy Designation

The Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ulixacaltamide (Praxis Precision Medicines, Boston, MA) for the treatment of people with essential tremor (ET). The designation was based on positive topline results from the Essential3 phase 3 clinical trial (NCT06087276) which showed that treatment with ulixacaltamide was associated with a significant improvement in function at week 8 as assessed by Modified Activities of Daily Living (mADL11) score (P<.0001).

Currently, propranolol is the only FDA-approved pharmacologic treatment for ET, with other therapies used off label. Ulixacaltamide is a highly selective small-molecule inhibitor of T-type calcium channels developed for the treatment of people with ET, the most common movement disorder. The agent is designed to reduce abnormal neuronal burst firing within the cerebello-thalamo-cortical circuit associated with tremor activity. In a statement from Praxis Precision Medicines, the company reported that a pre–New Drug Application (NDA) meeting with the FDA has been completed, and the company is aligned with the agency on the content of a future submission. Based on these interactions, Praxis Precision Medicines plans to submit an NDA for ulixacaltamide in early 2026, according to the company’s announcement.

The Breakthrough Therapy Designation was supported by data from the Essential3 program, which consisted of 2 pivotal phase 3 studies evaluating ulixacaltamide in individuals with ET. The FDA’s Breakthrough Therapy Designation program is reserved for investigational therapies for which preliminary clinical evidence suggests meaningful improvement on clinically significant endpoints compared with available therapies.

Source: Praxis Precision Medicines. FDA grants breakthrough therapy designation to ulixacaltamide for essential tremor. GlobeNewswire. Published December 29, 2025. Accessed December 29, 2025.