4 October 2018 - Orchard Therapeutics today announced that the EMA has granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta thalassaemia, the most severe form of beta thalassaemia.

PRIME designation for OTL-300 is based on data from preclinical and early clinical programs evaluating the autologous ex vivo gene therapy in transfusion-dependent beta thalassaemia patients, including data collected from nine patients in an ongoing proof-of-concept clinical trial of 10 patients currently being conducted by the San Raffaele-Telethon Institute for Gene Therapy. 

As of September 2018, OTL-300 has been evaluated in a total of nine patients. Of the seven patients with at least 12 months of follow-up as of April 2018, significant reductions in transfusion frequency and volume requirements were observed in five patients. Additionally, three of the four paediatric patients were transfusion-free since approximately one month post-treatment, and reductions in transfusion volume requirements were observed in two out of three adult patients, with one patient transfusion-free over a period of nine months. All patients are alive, and safety data from the nine patients treated in this clinical trial indicate OTL-300 was generally well-tolerated.

Read Orchard Therapeutics press release