A rare pediatric disease designation for its achondroplasia treatment and a subsequent $200 million private placement boosted shares of Tyra Biosciences Inc. on Feb. 2 by 29.3%.
Satellos Bioscience Inc. has received orphan drug designation and rare pediatric disease designation from the FDA for SAT-3153 for the potential treatment of Duchenne muscular dystrophy (DMD). The first-in-class oral small-molecule therapeutic is designed to restore the innate muscle regeneration process independent of dystrophin and regardless of exon mutation status.
The FDA has awarded rare pediatric disease designation to Orphagen Pharmaceuticals Inc.'s OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small-molecule antagonist to steroidogenic factor-1 (SF-1; NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
PERTH, Australia – The FDA granted Melbourne-based Neuren Pharmaceuticals Ltd. and San Diego, California-based Acadia Pharmaceuticals Inc. a rare pediatric disease designation for trofinetide for treatment of Rett syndrome, a rare neurodevelopmental congenital central nervous system disorder.
PERTH, Australia – The FDA granted Melbourne-based Neuren Pharmaceuticals Ltd. and San Diego, California-based Acadia Pharmaceuticals Inc. a rare pediatric disease designation for trofinetide for treatment of Rett syndrome, a rare neurodevelopmental congenital central nervous system disorder.
New York-based startup 3Dbio Therapeutics snagged a rare pediatric disease designation from the U.S. FDA for Aurinovo, its investigational combination product for reconstruction of the outer ear in patients with microtia. The product offers an alternative to established treatments, none of which enable patients to regain an auricle comprising living tissue other than rib grafts, which are associated with significant donor site morbidity.
