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Satellos Bioscience Receives Orphan Drug Designation from the U.S. FDA for SAT-3153, a First-in-Class Small Molecule Therapeutic for Duchenne Muscular Dystrophy
Company also recently received Rare Pediatric Disease Designation from the FDA for SAT-3153 for t...
About this update from Satellos Bioscience Inc
[{"type":"text","content":"Satellos Bioscience Receives Orphan Drug Designation from the U.S. FDA for SAT-3153, a First-in-Class Small Molecule Therapeutic for Duchenne Muscular DystrophyCompany also recently received Rare Pediatric Disease Designation from the FDA for SAT-3153 for the treatment of pediatric patients with DuchenneToronto, Ontario--(Newsfile Corp. - August 2, 2023) - Satellos Bioscience Inc. (TSXV: MSCL) (OTCQB: MSCLF) (\"Satellos\" or the \"Company\"), a drug discovery company developing small molecule therapeutics to regenerate muscle as a new approach to treating muscle diseases and disorders, announced today that that U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to SAT-3153 for the potential treatment of Duchenne muscular dystrophy (\"Duchenne\" or \"DMD\"). SAT-3153 is a first-in-class oral small molecule therapeutic designed to restore the innate muscle regeneration process independent of dystrophin and regardless of exon mutation status.\"Receiving orphan drug designation and rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating Duchenne,\" said Frank Gleeson, chief executive officer for Satellos. \"We continue to make important progress in our drug development program and IND-enabling studies, and we are focused on advancing SAT-3153 into the clinic next year.\"The FDA grants Orphan Drug Designation to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including the potential for a seven-year market exclusivity upon regulatory approval, exemption from FDA application fees, tax credits for qualified clinical trials, and a priority review voucher.The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, a sponsor who receives an approval for a drug or biologic for a \"rare pediatric disease\" may be eligi...