Published on 3 Apr 2024 on Zacks via Yahoo Finance
EMA Accepts Rocket (RCKT) Gene Therapy Filing for Fanconi Anemia
Rocket Pharmaceuticals RCKT announced that the EMA accepted the marketing authorization application (MAA), seeking approval for RP-L102, an investigational gene therapy for fanconi anemia (FA).
FA is a rare genetic disorder that mainly affects the bone marrow. It is also marked by cancer predisposition and congenital malformations.
The MAA acceptance is based on data from a phase I/II study wherein FA patients treated with RP-L102 achieved sustained genetic and comprehensive phenotypic corrections combined with hematologic stabilization. The therapy was well-tolerated in study participants.