1. PRAX plans to file a New Drug Application for relutrigine in early 2026. 2. Positive FDA discussions suggest a strong path towards NDA submission. 3. EMBOLD study shows significant improvement in seizures for patients. 4. Relutrigine has achieved multiple designations from regulatory authorities. 5. PRAX's therapy addresses critical needs in pediatric CNS disorders.
FAQ
Why Bullish?
The confirmation for NDA filing indicates progress in PRAX’s developmental pipeline. Historically, similar NDA filings have positively influenced stock prices for biopharmaceutical companies.
How important is it?
The article discusses upcoming FDA submissions that could indicate a breakthrough in treatment options.
Why Long Term?
The successful NDA submission could lead to eventual product launch, impacting revenues long-term, as seen in other drug approvals.
Praxis Precision Medicines to File NDA for Relutrigine to Treat SCN2A and SCN8A Encephalopathies
BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a biopharmaceutical company focused on developing therapies for central nervous system (CNS) disorders, has announced that it is set to file a New Drug Application (NDA) for its investigational therapy, relutrigine, in early 2026. This announcement follows successful discussions with the FDA regarding the application.
Significance of NDA Filing for Relutrigine
The anticipated NDA submission represents a significant milestone for Praxis, as the drug targets developmental and epileptic encephalopathies (DEEs) which severely affect young patients. Marcio Souza, president and CEO of Praxis, expressed optimism about the collaborative discussions with the FDA, stating, “Building on the unprecedented and compelling results from the EMBOLD study, we are advancing toward delivering a much-needed therapy for children living with these devastating diseases.”
About Relutrigine (PRAX-562)
Relutrigine is a pioneering small molecule that acts as a preferential inhibitor of persistent sodium current, a crucial mechanism linked to seizures in severe DEEs. Key features of relutrigine include:
- Demonstrated dose-dependent seizure inhibition in various animal models, including SCN2A and SCN8A.
- A well-tolerated safety profile established through three Phase 1 studies.
- Significant improvements in motor seizures reported during the Phase 2 EMBOLD study.
- Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for SCN2A-DEE, SCN8A-DEE, and Dravet syndrome.
- Breakthrough Therapy Designation from the European Medicines Agency for both SCN2A-DEE and SCN8A-DEE.
Clinical Impact and Future Implications
The data emerging from the EMBOLD study indicates that relutrigine has not only shown robust short- and long-term reductions in seizure frequency but has also maintained seizure freedom in some patients. These findings underscore the potential of relutrigine to transform the treatment landscape for children suffering from these debilitating conditions. As Praxis moves forward, stakeholders are eager to see the continuation of this positive trajectory towards regulatory approval and market availability.
About Praxis Precision Medicines
Praxis Precision Medicines is dedicated to decoding genetic insights to create therapies targeting CNS disorders caused by neuronal excitation-inhibition imbalance. Employing its innovative platforms, including Cerebrum™ and Solidus™, the company aims to address both rare and more prevalent neurological disorders. Currently, Praxis has a strong pipeline with several clinical-stage product candidates across movement disorders and epilepsy.
Forward-Looking Statements
This announcement contains forward-looking statements as per the Private Securities Litigation Reform Act of 1995, reflecting Praxis' expectations and plans related to the development of products, including future NDA submissions for relutrigine. These statements entail risks and uncertainties and should be considered speculative, given the variable nature of clinical trials and regulatory approvals.
For further details, visit www.praxismedicines.com, and stay updated by following Praxis on social media platforms including Facebook, Instagram, LinkedIn, and Twitter/X.