A Fanconi Anemia (FA) investigative gene therapy now has PRIority MEdicines (PRIME) designation from the EMA. This designation helps to accelerate the development of therapies for patient populations who have a high unmet need.
RP-L102 is a lentiviral vector created by Rocket Pharmaceuticals. It has now received all of the designations which support accelerated development from the European Union and the United States, showcasing the hope regulatory agencies have in this therapy. Additionally, the approval of these designations help improve the collaboration between agencies and researchers and aid the initiation of clinical trials.
The hope is that the culmination of these strategies will help treatments reach patients sooner.
Research Updates
The newest PRIME designation has come from data which has shown that the therapy has resulted in a durable engraftment. Trial participants have approached a mosaic phenotype of FA. In other words, RP-L102 could lead to phenotypic reversal.
A Phase 2 global trial of this therapy is still ongoing. The lead clinical sites are Lucile Packard Children’s Hospital Stanford and the Hospital Infantil Universitario Niño Jesús.
This trial will treat patients with RP-L102 “Process B” whereas initial studies treated patients with “Process A.” Process B utilizes RP-L102, transduction enhancers, and a modified stem cell enrichment process. The EMA and the FDA have collaborated on the primary endpoint of this trial, the presence of MMC-resistance. This is found in the bone marrow of cells and is utilized as a way of measuring phenotypic correction.
Researchers at the University of Minnesota are evaluating the bone marrow of patients. Additionally, the school is contributing to advisory activities.
You can read more about this therapy here.
