16 December 2019 - Milestone gives Fanconi anaemia program all accelerated regulatory tools in U.S. and EU, including FDA regenerative medicine advanced therapy, fast track and orphan designations.
Rocket Pharmaceuticals today announces that the EMA has granted PRIority MEdicines (PRIME) eligibility to RP-L102, the Company’s lentiviral vector-based gene therapy for the treatment of Fanconi Anaemia.
PRIME is a program launched by the EMA to enhance support for the development of medicines that target an unmet medical need.