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First targeted therapy for severe childhood epilepsy nears FDA approval

First targeted therapy for severe childhood epilepsy nears FDA approval

New Capabilities

Relutrigine could become the first FDA-approved treatment for SCN2A and SCN8A developmental epileptic encephalopathies, with a decision expected by September 2026

March 30th, 2026: FDA accepts application with priority review

Overview

Updated May 30

Children with SCN2A and SCN8A developmental and epileptic encephalopathies have never had a drug designed for their condition, cycling through four or more unapproved medications as seizures persist and development regresses. The FDA accepted Praxis Precision Medicines' application for relutrigine and granted priority review, with a decision expected by September 27, 2026.

The clinical evidence was strong enough that an independent safety committee told researchers to stop the trial early—the drug was already working. In the registrational study, patients on relutrigine had a 53% greater seizure reduction compared to placebo, with 66% more seizure-free days. If approved, it would be the first precision therapy for these conditions, and thousands of children worldwide would finally have a treatment designed specifically for their rare genetic epilepsy.

Why it matters

Thousands of children with devastating genetic epilepsy may soon have the first drug ever designed specifically for their condition.

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Key Indicators

53%
Placebo-adjusted seizure reduction
In the registrational study, relutrigine reduced countable motor seizures by 53% more than placebo over 16 weeks.
0
Current FDA-approved targeted therapies
No targeted treatments exist today for SCN2A or SCN8A developmental and epileptic encephalopathies.
~90%
Seizure reduction at 11 months
Patients in the open-label extension phase saw approximately 90% seizure reduction after 11 months on the drug.
Sep 27, 2026
FDA target decision date
The agency will decide whether to approve relutrigine by this date under its six-month priority review timeline.
$7.7B
Praxis market capitalization
The company's valuation reflects investor confidence across four late-stage clinical programs.

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People Involved

Marcio Souza
Marcio Souza
President and Chief Executive Officer, Praxis Precision Medicines
Leading company through transition to commercial-stage
 Steven Petrou
Steven Petrou
Co-Founder, President of Research & Development, and Chief Scientific Officer, Praxis Precision Medicines
Leading R&D across four clinical-stage programs

Organizations Involved

Praxis Precision Medicines
Praxis Precision Medicines
Biopharmaceutical Company
Transitioning from clinical-stage to commercial-stage with two NDA submissions under FDA review

A Boston-based precision neuroscience company developing targeted therapies for neurological disorders caused by imbalances in neuronal excitation and inhibition.
U.S. Food and Drug Administration
U.S. Food and Drug Administration
Federal regulatory agency
Reviewing relutrigine NDA under priority review

The federal agency responsible for evaluating and approving new drugs, including granting expedited pathways for therapies addressing serious conditions with unmet medical needs.
FamilieSCN2A Foundation
FamilieSCN2A Foundation
Patient Advocacy Organization
Leading advocacy for SCN2A disorder research and treatment access

A nonprofit dedicated to funding research and supporting families affected by SCN2A-related disorders, working directly with the FDA on patient-centered drug development.

Timeline

September 2015 March 2026

7 events Latest: March 30th, 2026 · 5 months ago
Tap a bar to jump to that date

  1. FDA accepts application with priority review

    Latest Regulatory

    The FDA accepted the relutrigine NDA and granted priority review, compressing the review timeline from ten months to six. Decision expected by September 27, 2026.

  2. Praxis submits new drug application to the FDA

    Regulatory

    Praxis filed its first NDA, seeking approval for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies.

  3. Results presented at American Epilepsy Society meeting

    Scientific

    Full EMBOLD data presented to the epilepsy research community at the AES Annual Meeting.

  4. Trial stopped early—drug already working

    Clinical Trial

    An independent Data Monitoring Committee recommended halting the EMBOLD registrational study early after relutrigine demonstrated clear efficacy: 53% placebo-adjusted seizure reduction and 66% increase in seizure-free days.

  5. FDA grants Breakthrough Therapy designation

    Regulatory

    The FDA recognized relutrigine's potential to offer substantial improvement over existing therapies for SCN2A and SCN8A epileptic encephalopathies.

  6. EMBOLD Cohort 1 shows 46% seizure reduction

    Clinical Trial

    First cohort of 16 patients showed a 46% placebo-adjusted reduction in motor seizures, with five patients achieving seizure-free status versus none on placebo.

  7. Praxis Precision Medicines founded

    Corporate

    Kiran Reddy, David Goldstein, and Steven Petrou founded the company to translate genetic epilepsy research into precision therapies.

Historical Context

3 moments from history that rhyme with this story — and how they unfolded.

April 2017

Brineura approval for CLN2 disease (2017)

BioMarin won FDA approval for Brineura (cerliponase alfa), the first treatment for neuronal ceroid lipofuscinosis type 2 (CLN2 disease), a fatal neurodegenerative condition that strikes children around age two and causes seizures, loss of motor function, and death by early adolescence. The approval was based on a study of just 24 patients—even smaller than relutrigine's EMBOLD trial.

Then

Brineura was approved based on a single-arm study with a historical comparator, demonstrating the FDA's willingness to accept unconventional trial designs for devastating pediatric diseases. BioMarin received a Priority Review Voucher it sold to Gilead for $125 million.

Now

The approval established precedent that small trials in ultra-rare pediatric diseases can support approval when the unmet need is severe and the treatment effect is clear.

Why this matters now

Relutrigine's EMBOLD trial enrolled 51 patients—small by conventional standards but larger than Brineura's pivotal study. The Brineura precedent suggests the FDA is prepared to approve based on limited patient numbers when the disease is severe, no alternatives exist, and the clinical signal is strong.

June 2018

Epidiolex approval for Dravet and Lennox-Gastaut syndromes (2018)

GW Pharmaceuticals won FDA approval for Epidiolex (cannabidiol), the first plant-derived cannabinoid medicine and the first drug approved specifically for Dravet syndrome and Lennox-Gastaut syndrome—severe childhood epilepsies that, like SCN2A and SCN8A encephalopathies, had limited targeted treatment options. The approval followed a Breakthrough Therapy designation and Priority Review.

Then

Epidiolex provided the first FDA-approved option for two devastating epilepsy syndromes, generating over $500 million in annual revenue within two years.

Now

The approval opened a pathway for precision therapies targeting specific epilepsy subtypes and demonstrated that the FDA would support accelerated review for drugs addressing severe childhood neurological conditions with strong clinical evidence.

Why this matters now

Relutrigine follows a nearly identical regulatory trajectory—Breakthrough Therapy, Orphan Drug, and Priority Review designations for a severe pediatric epilepsy with no approved targeted therapy. Epidiolex's commercial success demonstrated robust demand among families desperate for effective treatment options.

May 2019

Zolgensma approval for spinal muscular atrophy (2019)

Novartis (through its AveXis subsidiary) won FDA approval for Zolgensma, a one-time gene therapy for spinal muscular atrophy (SMA) in children under two years old. The drug earned Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations—the same trio relutrigine holds. Priced at $2.1 million per patient, it became the most expensive drug in the world.

Then

Zolgensma generated over $1.3 billion in its first full year of sales. AveXis received a Rare Pediatric Disease Priority Review Voucher, which Novartis sold to Takeda for $350 million.

Now

The approval validated the commercial model for precision therapies in ultra-rare pediatric diseases and demonstrated the value of Priority Review Vouchers as a financial incentive for rare disease drug development.

Why this matters now

Praxis holds the same Rare Pediatric Disease designation and could receive a Priority Review Voucher worth hundreds of millions upon approval. Zolgensma's trajectory illustrates how targeted therapies for rare childhood conditions can generate substantial revenue despite small patient populations—a model Praxis is positioning relutrigine to follow.

Sources

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GlobeNewsWire MarketScreener Praxis Precision Medicines Investor Relations BioSpace NeurologyLive GlobeNewsWire