Praxis Aligns With FDA On Relutrigine Program For SCN2A And SCN8A Epilepsies Ahead Of Phase 3 Milestones

Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company developing therapies for central nervous system (CNS) disorders, announced that it has reached alignment with the U.S. FDA on several aspects of its relutrigine program for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The FDA agreed that the ongoing EMBOLD study could serve as the primary evidence of effectiveness for the company’s New Drug Application (NDA). If the interim analysis planned for Q4 2025 is positive, Praxis aims to submit the NDA in early 2026.

Chief Scientific Officer Steven Petrou stated that the breakthrough therapy designation (BTD) enabled productive discussions with the FDA, helping expedite relutrigine’s potential path to approval. Upon positive results from the interim analysis, Praxis intends to move quickly toward the NDA submission, which could become the company’s second NDA under review, following ulixacaltamide.

SCN2A- and SCN8A-DEEs are rare pediatric epilepsies marked by early-onset, drug-resistant seizures, severe neurological decline, and high mortality rates. No FDA-approved therapies currently exist, and available treatments often involve off-label use of anti-seizure medications with limited efficacy and safety concerns. The EMBOLD study is designed as a 16-week randomized, placebo-controlled trial evaluating seizure frequency reduction with relutrigine, while the ongoing EMERALD study will support future expansion across broader DEE populations, potentially leading to an sNDA by 2027.