This section highlights FDA-related milestones and regulatory updates for drugs developed by Praxis Precision Medicines (PRAX).
Over the past two years, Praxis Precision Medicines has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
PRAX-562, PRAX-562, and Ulixacaltamide. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
PRAX-562 (SCN2A) FDA Regulatory Events
PRAX-562 (SCN2A) is a drug developed by Praxis Precision Medicines for the following indication: CN2A development and epileptic encephalopathy (SCN2A-DE.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- PRAX-562 (SCN2A)
- Announced Date:
- July 17, 2025
- Indication:
- CN2A development and epileptic encephalopathy (SCN2A-DE
Announcement
Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted BTD for relutrigine, a sodium channel functional state modulator for pediatric use for the treatment of patients with SCN2A and SCN8A DEEs.
AI Summary
Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for relutrigine, a sodium channel functional state modulator. This designation is for its use in pediatric patients suffering from SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The decision was based on highly promising results from the Phase 2 EMBOLD trial, where relutrigine demonstrated impressive seizure reduction and extended seizure-free periods in a particularly severe patient group.
The BTD is an important milestone, as it speeds up the development and review process for treatments showing significant improvements over current therapies. This development supports Praxis’s commitment to fast-track effective therapies for children with these challenging epileptic conditions, offering hope for better management and improved quality of life for patients and their families.
Read Announcement- Drug:
- PRAX-562 (SCN2A)
- Announced Date:
- September 2, 2024
- Indication:
- CN2A development and epileptic encephalopathy (SCN2A-DE
Announcement
Praxis Precision Medicines announced that it will share the topline results from the Phase 2 EMBOLD study of relutrigine (PRAX-562) in SCN2A,
AI Summary
Praxis Precision Medicines announced that it will share the topline results from the Phase 2 EMBOLD study of relutrigine (PRAX-562) focused on patients with SCN2A-related conditions. The study is part of the company’s efforts to explore potential treatments for severe developmental epileptic encephalopathies (DEEs) caused by mutations in SCN2A and SCN8A. The topline data will be released on Tuesday, September 3, 2024, just before the market opens. After the announcement, Praxis will host a conference call and webcast at 8:00 a.m. ET to discuss the trial results. This update is a key step in Praxis’s mission to translate genetic findings into therapies aimed at restoring the balance between neuronal excitation and inhibition in the brain, potentially offering new hope for patients with difficult-to-treat CNS disorders.
Read Announcement
PRAX-562 (SCN8A) FDA Regulatory Events
PRAX-562 (SCN8A) is a drug developed by Praxis Precision Medicines for the following indication: SCN8A development and epileptic encephalopathy (SCN8A-DEE).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- PRAX-562 (SCN8A)
- Announced Date:
- July 17, 2025
- Indication:
- SCN8A development and epileptic encephalopathy (SCN8A-DEE)
Announcement
Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted BTD for relutrigine, a sodium channel functional state modulator for pediatric use for the treatment of patients with SCN2A and SCN8A DEEs.
AI Summary
Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for relutrigine. This drug is a sodium channel functional state modulator being developed for pediatric patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The designation was supported by promising data from the Phase 2 EMBOLD trial. In this study, patients with severe DEEs, who had not responded to multiple previous treatments, experienced significant clinical improvements. Early results showed an average 90% reduction in seizures and longer seizure-free intervals, offering hope for better quality of life for patients and their caregivers.
The BTD allows for expedited development and regulatory review. Praxis is planning further studies, including the ongoing registrational EMBOLD cohort 2 and the EMERALD study, to confirm the benefits of relutrigine in treating these rare and challenging conditions.
Read Announcement- Drug:
- PRAX-562 (SCN8A)
- Announced Date:
- September 2, 2024
- Indication:
- SCN8A development and epileptic encephalopathy (SCN8A-DEE)
Announcement
Praxis Precision Medicines announced that it will share the topline results from the Phase 2 EMBOLD study of relutrigine (PRAX-562) in SCN8A
AI Summary
Praxis Precision Medicines announced that it will share the topline results from its Phase 2 EMBOLD study evaluating relutrigine (PRAX-562) in patients with SCN8A Developmental and Epileptic Encephalopathies (DEEs). The study examines the potential of relutrigine to address imbalances in neuronal excitation and inhibition, which are central to these severe neurological disorders. This announcement is scheduled for Tuesday, September 3, 2024, before the market opens, with further details to be provided during a conference call and webcast at 8:00 a.m. ET.
The information from the study is eagerly anticipated as researchers and investors look forward to understanding how relutrigine may benefit patients with SCN8A-associated conditions. The initiative highlights Praxis Precision Medicines' commitment to translating genetic insights into treatments for central nervous system disorders and improving care for those affected by these challenging conditions.
Read Announcement
Ulixacaltamide Essential1 FDA Regulatory Events
Ulixacaltamide Essential1 is a drug developed by Praxis Precision Medicines for the following indication: Designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity and being developed for Essential Tremor.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Ulixacaltamide Essential1
- Announced Date:
- September 26, 2024
- Indication:
- Designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity and being developed for Essential Tremor.
Announcement
Praxis Precision Medicines, Inc. announced the presentation of a poster and platform talk showcasing its Phase 3 clinical program evaluating ulixacaltamide for adults with essential tremor (ET) at the Movement Disorders Society (MDS) 2024 International Congress taking place in Philadelphia, Pennsylvania, September 27 – October 1, 2024.
AI Summary
Praxis Precision Medicines announced that it will present a poster and give a platform talk at the Movement Disorders Society (MDS) 2024 International Congress. The event will be held in Philadelphia, Pennsylvania, from September 27 to October 1, 2024. The presentations will focus on the company’s Phase 3 clinical program evaluating ulixacaltamide, a new therapy for adults with essential tremor (ET). Ulixacaltamide is designed to work by blocking abnormal neuronal burst firing in the brain, aiming to reduce tremor activity. The platform talk and poster presentation will share important progress and insights from the clinical program, and attendees can learn more about the science behind the therapy. This opportunity highlights Praxis Precision Medicines' dedication to developing treatments for central nervous system disorders and advancing innovative solutions to improve patient quality of life.
Read Announcement
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