Biotech News

Praxis Precision Medicines Receives FDA Breakthrough Therapy Designation for Elsunersen for the Treatment of Seizures Associated with SCN2A Developmental and Epileptic Encephalopathy Caused by Gain of Function Variants

Praxis Precision Medicines (PRAX)Praxis Precision MedicinesFDA / regulatory

The Breakthrough Therapy Designation (BTD) was granted based on the positive results from the EMBRAVE Part A trial of elsunersen Elsunersen demonstrated a 77% sham-adjusted reduction in monthly seizures (p=0.015), with benefit sustained in the open-label extension for up to one year The pivotal

Related news

PRAXPraxis Precision Medicines2026-06-29 20:30 EST

Praxis Precision Medicines Announces Extension Period for Relutrigine for Treatment of SCN2A and SCN8A Developmental and Epileptic Encephalopathies

IKTInhibikase Therapeutics2026-07-23 12:00 EST

Inhibikase Therapeutics Announces FDA Orphan Drug Designation Granted to IKT-001 for the Treatment of PAH

KTTAPasithea Therapeutics2026-06-02 11:02 EST

Pasithea Therapeutics Announces Orphan Drug Designation by FDA of PAS-004 for Treatment of Amyotrophic Lateral Sclerosis (ALS)

VYGRVoyager Therapeutics2026-06-01 12:00 EST

Voyager Receives FDA IND Clearance for VY1706, First Gene Therapy Approach to Reducing Tau Production in the Brain for Alzheimer’s Disease

PRAXPraxis Precision Medicines2026-07-07 12:00 EST

Praxis Precision Medicines and Remagine Labs Announce Strategic Collaboration to Develop Transdermal Ulixacaltamide for Essential Tremor

GANXGain Therapeutics2026-06-29 20:05 EST

Gain Therapeutics Receives FDA Clearance of IND to Advance GT-02287 into Phase 2 Clinical Development for Parkinson’s Disease

AXSMAxsome2026-07-15 11:00 EST

Axsome Therapeutics Announces FDA Acceptance of New Drug Application for AXS-12 for the Treatment of Cataplexy in Narcolepsy

CGTXCognition Therapeutics2026-06-24 11:30 EST

Cognition Therapeutics Aligns on Key Aspects of Pivotal Study for Zervimesine (CT1812) in DLB Psychosis Following Receipt of FDA Meeting Minutes

BCLIBrainstorm Cell Therapeutics Inc.2026-07-28 12:00 EST

BrainStorm Appoints Former FDA Associate Commissioner Peter J. Pitts as Executive Chairman

CGTXCognition Therapeutics2026-05-21 11:30 EST

Cognition Therapeutics Completes Meeting with FDA for Zervimesine (CT1812) in Dementia with Lewy Bodies with Psychosis

Praxis Precision Medicines

Precision Neuroscience Praxis Precision Medicines develops therapies for genetically and physiologically defined disorders of neuronal excitation. Its pipeline spans movement disorders and developmental epilepsies. Ulixacaltamide Ulixacaltamide is a small-molecule T-type calcium-channel modulator under FDA review for essential...

Recent company news

Stock data

Close
370.16 USD
Market cap
7.19B USD
Exchange
NASDAQ
Sector
Biopharma
Location
United States