Posted by Michael Wonder on 30 May 2019
Fibrocell receives FDA regenerative medicine advanced therapy designation for FCX-007 gene therapy for the treatment of RDEB
29 May 2019 - Fibrocell Science today announced that the U.S. FDA has granted the regenerative medicine advanced therapy designation to FCX-007, the Company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa—a devastating, genetic skin disease with high mortality.
The Company expects to initiate the Phase 3 clinical trial for FCX-007 in the second quarter of 2019. Fibrocell projects enrollment and dosing of Phase 3 patients will be completed in the third quarter of 2020 and data collection for the primary endpoint will be completed in the fourth quarter of 2020. If the Phase 3 clinical trial is successful and completed within the projected timeframe, Fibrocell expects to file a biologics license application for FCX-007 in 2021.
The RMAT designation augments the orphan drug, rare paediatric disease and fast track designations previously granted to FCX-007 by the FDA.
