Praxis Precision Medicines, Inc. Receives Rare Pediatric Disease and Orphan Drug Designations for Severe Pediatric Epilepsy Programs
January 07, 2021 at 06:30 pm IST
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Praxis Precision Medicines, Inc. 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). 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 FDA’s rare pediatric disease designation program is designed for serious and life-threatening diseases primarily affecting children under the age of 18 with fewer than 200,000 people in the United States. Under the FDA’s rare pediatric disease and voucher programs, subject to FDA approval of a product with such designation, a company may be eligible to receive a priority review voucher that can be redeemed to obtain priority review for any subsequent marketing application or sold or transferred to other companies. The FDA’s orphan drug designation program is designed to encourage and facilitate the development of investigational treatments for rare diseases that affect fewer than 200,000 people in the United States. The designation provides various development and commercial incentives, including tax credits for clinical research costs, waiver or partial payment of application fees and market exclusivity for seven years following FDA approval.
Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company. It is focused on translating insights from genetic epilepsies into the development of therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. It is applying genetic insights to the discovery and development of therapies for neurological disorders through two platforms, using its understanding of shared biological targets and circuits in the brain. Its platform includes Cerebrum, a small molecule platform, which utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies, and Solidus, is an antisense oligonucleotide platform, is an efficient, targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology. Its product candidates include ulixacaltamide, PRAX-562, PRAX-628 and elsunersen.
PRAXIS PRECISION MEDICINES, INC. 
