Rocket Pharmaceuticals, a late-stage biotechnology company, has announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for RP-L102, its gene therapy for Fanconi Anemia (FA). The EMA’s acceptance marks significant progress in the company’s aim to bring this potential treatment to patients affected by this rare genetic disorder.
FA is caused by mutations in the FANCA gene that affect DNA repair, and it is characterized by bone marrow failure, cancer predisposition, and congenital malformations. There are currently no available options to potentially prevent bone marrow failure for FA patients.
The MAA acceptance was based on data from the global RP-L102 Phase 1/2 clinical trial, which demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization. The treatment, administered without cytotoxic conditioning, was well tolerated, and there were no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to RP-L102.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.