Section 3 of 6
Product information and disease description
64 evidence topics · 60 sources
Product description
Phase of product development
Breakthrough Therapy Designation
| Type of information | Source quotation |
|---|---|
| Special FDA designation: date | “December 29, 2025” |
| Special FDA designation | “Breakthrough Therapy Designation for Ulixacaltamide HCl in Essential Tremor” |
Regulatory submission: NDA submitted by February 2026
Regulatory review and FDA Advisory Committee meeting
Launch
No evidence found.
Approval dates in other countries
No evidence found.
Product information
Generic, brand name and therapeutic class of product
Manufacturer and investigational product name
| Type of information | Source quotation |
|---|---|
| Manufacturer name | “Praxis Precision Medicines” |
| Unapproved product name | “Ulixacaltamide HCl” |
Manufacturer: Praxis Precision Medicines
Confirmed commercial brand name
No evidence found.
Dosage forms and strengths
Investigational formulation in PRAX-944-221
| Attribute | Quoted protocol information |
|---|---|
| Dosage form | “Modified-release tablet” |
| Unit strength | “20 mg” |
| Route | “Oral” |
Commercial dosage forms and strengths
No evidence found.
Material safety data sheet
No evidence found.
Average sales price and wholesale acquisition cost
Manufacturer pricing discussion without a disclosed final price
Marketed-product average sales price and wholesale acquisition cost
Not applicable.
American hospital formulary service (AHFS), or other drug classification
AHFS classification code
No evidence found.
Indication
Investigational indication
| Type of information | Source quotation |
|---|---|
| Disease or anticipated indication | “Essential Tremor” |
Pharmacology
Mechanism of action
Structural pharmacology of Z944 and Cav3.1
Pharmacodynamics
Preclinical absence-seizure model
Experimental Cav1.4 channel modulation
Pharmacokinetics
Steady-state sampling in the healthy-participant study
Numerical absorption, distribution, metabolism, and elimination parameters
No evidence found.
Contraindications/Warnings/Precautions/Adverse effects
Warnings and precautions
Not applicable.
Special populations
Adult age range in the original Essential3 eligibility criteria
Pediatric use, pregnancy, lactation, and renal or hepatic impairment
No evidence found.
Drug/Drug, drug/disease interactions
Effects of other drugs on Ulixacaltamide
No evidence found.
Effects of Ulixacaltamide on other drugs
No evidence found.
Dosing and administration
Dosage
Essential3 investigational regimen
Administration
Access and distribution
Anticipated patient support programs
No evidence found.
Co-prescribed/Concomitant therapies
Stable background propranolol in Essential1
Effect of Ulixacaltamide on quality measures
No evidence found.
Product comparison
Head-to-head comparisons with established essential tremor therapies
No evidence found.
Placebo-controlled study of NBI-827104
Adverse events in the SAGE-324 KINETIC trial
Place of product in therapy
Disease description
Definition and etiology
Epidemiology
Incidence of Essential tremor
Annual incidence in Rochester, Minnesota, 1935-79
Prevalence of Essential tremor
Prevalence by ascertainment strategy in northeast-central Pennsylvania
| Ascertainment strategy | Crude prevalence | Age-standardized prevalence |
|---|---|---|
| Neurology cohort | “0.261% (95% CI, 0.252–0.271)” | “0.40% (95% CI, 0.39–0.41)” |
| All-specialties cohort | “0.653% (95% CI, 0.638–0.668)” | “1.01% (95% CI, 1.00–1.02)” |
Natural history, survival, and mortality
Historical population-based survival comparison
Pathophysiology
T-type calcium channels and aberrant neuronal burst firing in essential tremor and Parkinson’s disease
Diagnosis
Functional assessment in the current clinical advisory
Clinical presentation - signs and symptoms
Motor symptoms: upper limb tremor and additional affected regions
Nonmotor symptoms: cognition, mood, balance, and sleep
Long-term morbidity
Burden of Essential tremor
Humanistic burden and health-related quality of life
Disease-specific quality-of-life instrument validation
Economic burden and healthcare resource utilization
All-cause and essential tremor-related healthcare costs in 2019 US dollars
Economic impact of Essential tremor on families
Care partner prevalence and weekly caregiving time
Unpaid caregiving excluded from medical-cost estimates
Activities supported by informal caregivers
Economic impact of diagnostic testing
Not applicable.
Approaches to treatment
Current treatment options and standard of care
Beta-Adrenergic blockers
Established first-line pharmacologic recommendations
Antiseizure medications
Primidone in the MDS evidence-based review
Topiramate dose qualification in the MDS review
Benzodiazepines
Second-line pharmacologic recommendations including alprazolam
Botulinum toxin injections
Recommendations for refractory essential tremor
Deep brain stimulation
Ablative thalamic procedures
Radiofrequency and MRI-guided focused ultrasound thalamotomy
Peripheral nerve stimulation
Randomized trial of transcutaneous peripheral nerve stimulation
| Outcome | Active stimulation | Sham stimulation |
|---|---|---|
| Randomized participants | “83” | “42” |
| mADL reduction at 90 days | “6.9 points” | “2.7 points” |
| Skin irritation | “28 of 83 participants (33.7%)” | “2 of 42 (4.8%)” |
Adaptive equipment and occupational strategies
Assistive devices in the current clinical advisory
Limitations of current therapies
Summary
At two years, reported treatment discontinuation was 40% for propranolol and 47% for primidone. A 2024 survey included 411 participants with essential tremor and 152 neurologists; up to 77% of surveyed participants reported that symptoms were not manageable with current treatments. Approximately half of participants in a separate claims-based essential tremor population received no pharmacotherapy during follow-up. The 2026 MDS review reported insufficient evidence for a confident efficacy recommendation under its review criteria. That finding concerns the certainty of the included evidence, rather than demonstrating that every intervention is ineffective.
Evidence gaps identified by the MDS evidence-based review
Updated evidence-based review of commercially available interventions
Place in treatment, anticipated use, and care setting
Summary
Ulixacaltamide is an investigational oral treatment for adults with essential tremor. The phase 3 regimen was 60 mg once daily after titration. Supporting evidence includes treatment alongside stable propranolol, but these data do not establish superiority to propranolol or primidone. The available pivotal studies compare ulixacaltamide with placebo, including withdrawal of treatment in selected responders. Routine use and final prescribing conditions remain dependent on regulatory review. The manufacturer reports an established distribution network; expanded-access requests must come from the treating physician and are considered individually.
Heterogeneity of treatment effect
Care management intervention strategies
Patient and caregiver psychosocial support
Other product development or post-marketing obligations required by the FDA
Not applicable.
Ongoing post-approval monitoring
Not applicable.
Expected outcomes of therapy
Daily-function outcome assessed in Essential3 Study 1
Disease modification, prevention of progression, and survival benefit
No evidence found.