Tag: fda orphan drug

Oral Infigratinib Shows Benefits Beyond Growth in Children with Achondroplasia
Clinical Insights & Research Pediatric Health Pharmacological Sleep Apnea

Oral Infigratinib Shows Benefits Beyond Growth in Children with Achondroplasia

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 […]

Idiopathic Hypersomnia Industry News Narcolepsy Pharmacological

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 […]