Business
Praxis Precision Medicines Receives Rare Pediatric Disease and Orphan Drug Designations for Severe Pediatric Epilepsy Programs
FDA grants RPD and ODD for PRAX-222 for treatment of SCN2A-DEE FDA grants RPD for PRAX-562 for treatment of SCN2A-DEE and SCN8A-DEE CAMBRIDGE, Mass., Jan. 07,
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"FDA grants RPD and ODD for PRAX-222 for treatment of SCN2A-DEE FDA grants RPD for PRAX-562 for treatment of SCN2A-DEE and SCN8A-DEE CAMBRIDGE, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal imbalance, today announced that the U.S. Food and Drug Administration (FDA) has granted both rare pediatric disease and orphan drug designations for PRAX-222 for the treatment of SCN2A developmental and epileptic encephalopathy (SCN2A-DEE). The FDA has also granted rare pediatric disease designation for PRAX-562 for the treatment of SCN2A-DEE and for the treatment of SCN8A developmental and epileptic encephalopathy (SCN8A-DEE). “As a company deeply rooted in the genetic drivers of severe pediatric epilepsies, these designations are especially meaningful and validating,” said Marcio Souza, president and chief executive officer of Praxis. “We are committed to drug development for both common and rare CNS diseases and look forward to progressing PRAX-562 and PRAX-222 in the clinic and ultimately to children who are in dire need of new treatment options.” PRAX-562 is a selective small molecule and is the first persistent sodium current blocker in development for the treatment of a wide range of rare CNS disorders. The clinical development plan for PRAX-562 encompasses exploring the broad potential for the mechanism of action in rare diseases through proof-of-concept trials in two rare types of cephalgia, and then expanding into a range of rare pediatric DEEs, including SCN8A-DEE and SCN2A-DEE. PRAX-562 is currently being evaluated in a Phase 1 clinical trial in adult healthy volunteers. PRAX-222 is an antisense oligonucleotide that is designed to lower the expression levels of the protein encoded by the SCN2A gene in patients with SCN2A gain-of-function epilepsy. The program is ongoing under a three-way collaboration with Ionis Pharmaceuticals, Inc. and RogCon, Inc. PRAX-222 is currently being evaluated in IND-enabling studies. “The potential broad utility of PRAX-562 in DEEs and other rare CNS disorders and the precision therapy approach of PRAX-222 represent two differentiated and potentially complementary treatment paradigms insp...