Keeping Track: Novel Orphan Drugs Amondys 45, Nulibry Clear US FDA; First-Line NSCLC Claim For Libtayo

The US Food Drug Administration closed out February with the approval of two novel therapies for rare diseases – Origin Biosciences’ Nulibry (fosdenopterin) for the genetic metabolic disorder Molybdenum Cofactor Deficiency Type A and Sarepta Therapeutics, Inc.’s Amondys 45 (casimersen) for selected Duchenne muscular dystrophy patients.

The approvals bring the Center for Drug Evaluation and Research’s novel agent approvals count for 2021 to nine