BridgeBio Pharma, Inc., presented new exploratory analyses from PROPEL 3, the global Phase 3 pivotal study of oral infigratinib in children living with achondroplasia, showing directionally favorable trends beyond growth across medical complications associated with achondroplasia, including sleep apnea, otitis media, and body composition. These data were presented at the […]
Tag: fda orphan drug
Avadel’s LUMRYZ Receives FDA Orphan Drug Designation for Idiopathic Hypersomnia
Avadel Pharmaceuticals plc, a biopharmaceutical company focused on transforming medicines to transform lives, announced that LUMRYZTM has been granted Orphan Drug Designation (ODD) from the U.S. Food & Drug Administration (FDA) for the treatment of Idiopathic Hypersomnia (IH). Specifically, ODD was granted based on the plausible hypothesis that LUMRYZ may be […]
Avadel Pharmaceuticals Receives FDA Approval of Bedtime Narcolepsy Treatment
Avadel Pharmaceuticals plc, announced that the U.S. Food & Drug Administration (FDA) has granted final approval to LUMRYZ, an extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. With final approval, LUMRYZ becomes the […]






