Also known as: developmental epilepsies
Developmental and epileptic encephalopathies (DEEs) are severe epilepsy syndromes that usually begin early in life and involve both frequent epileptic activity and developmental impairment. Neurologic and cognitive outcomes are often poor, and many forms are drug resistant. DEEs are considered among the most severe epileptic syndromes.
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
The FDA has accepted Praxis Precision Medicines' NDA for relutrigine with priority review and set a PDUFA target date of September 27, 2026. The therapy targets SCN2A and SCN8A developmental and epileptic encephalopathies and could become the first approved treatment for these rare conditions. The company has bolstered its cash position to support commercial launch preparations.
Praxis Precision Medicines submitted NDAs for ulixacaltamide and relutrigine to the FDA, reported cash and investments of $926 million as of December 31, 2025, and disclosed a net loss of $303.3 million for full-year 2025.
Praxis Precision Medicines has submitted new drug applications for ulixacaltamide in essential tremor and relutrigine in rare epilepsies, both granted Breakthrough Therapy Designation. The company reported $926 million in cash as of December 31, 2025.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07585643 |
IBIS - Investigating Reliability of BIS and SEDLINE Monitoring in Children With Developmental and Epileptic Encephalopathies (DEE). |
NOT_YET_RECRUITING | |
| NCT07531511 |
SLC6A1-NDD Prospective Longitudinal Natural History Study |
NOT_YET_RECRUITING | |
| NCT06700811 |
Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies |
RECRUITING | PHASE1 |
| NCT06149663 |
Intermediate-Size Expanded Access Protocol (EAP) for LP352 |
AVAILABLE |
