Rocket announces EMA accepted MAA for RP-L102 lentiviral

Rocket Pharmaceuticals announced that the European Medicines Agency, or EMA, accepted the Marketing Authorization Application, or MAA, for RP-L102, its lentiviral, or LV, vector-based investigational gene therapy for Fanconi Anemia, or FA, complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure, or BMF, cancer predisposition, and congenital malformations. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial. RP-L102 demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization. The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated. There were no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to RP-L102. The Biologics License Application for FA remains on track for submission to the FDA in the first half of 2024.