FDA’s drug center smashes its past records for new molecular entities and therapeutic biologic approvals; with five novel biologics approved by FDA’s biologics center, the agency’s novel approvals reach a stunning total of 64.
More than 90% of the US FDA’s Center for Drug Evaluation and Research actions on applications for novel agents were approvals in 2018, carrying the agency to its largest annual approval count since the modern era of the FDA was initiated by Prescription Drug User Fee Act of 1992.
CDER is known to have acted on 65 applications for new molecular entities and novel therapeutic biologics in 2018, resulting in 59
Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
