Orchard Therapeutics announce that the European Medicines Agency (EMA) has granted an investigational thalassemia drug OTL-300 Priority Medicines designation.
OTL-300, is an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia (TDBT), the most severe form of beta-thalassemia.
The PRIME program aims to increase regulatory support for the development of therapeutics targeting areas of high unmet need. PRIME designation provides enhanced interaction with the EMA to both optimise development and accelerate evaluations of applications. To be accepted for PRIME, an investigational therapy must demonstrate potential therapeutic advantage over available treatment options or benefit patients with no current treatments.
The OTL-300 Priority Medicines designation is based on data from preclinical and early clinical programs evaluating the autologous ex vivo gene therapy in TDBT 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. Safety data from the nine patients treated in this clinical trial indicate OTL-300 was generally well-tolerated.
TDBT is a rare inherited blood disorder caused by one of over 200 mutations in the HBB gene (or beta-globin gene). TDBT is characterised by failure to grow and gain weight, infection, and life-threatening anaemia, which occur within the first two years of life. The only curative approach is allogeneic hematopoietic stem cell transplantation which carries significant transplant-related morbidity and mortality. Most patients with TDBT undergo chronic blood transfusions due to the severity of their symptoms and, as a result, often develop side effects such as iron overload, which requires additional iron chelation therapy, leading to further serious side effects and reduced quality of life.
Orchard is dedicated to transforming the lives of patients with rare diseases through innovative gene therapies and recently raised $150 million to advance gene therapy pipeline.
Giuliana Ferrari, who leads the thalassemia gene therapy project at the San Raffaele- Telethon Institute for Gene Therapy said, “We are encouraged by the early clinical trial data in TDBT patients, which was started in 2015 in hopes of extending the hematopoietic stem cell gene therapy approach that has been so successful in such conditions as adenosine deaminase severe combined immunodeficiency (ADA-SCID), Wiskott–Aldrich syndrome (WAS) and Metachromatic Leukodystrophy (MLD) to other diseases with unmet medical needs such as TDBT. Hematopoietic stem cell gene therapy could offer a promising alternative to the current standards of care for adults and children living with TDBT.”
Mark Rothera, president and chief executive officer of Orchard said “We are pleased that the EMA has granted OTL-300 Priority Medicines designation based on encouraging preliminary results from an ongoing clinical trial that utilizes our ex vivo lentiviral gene therapy approach to treat the underlying genetic cause of TDBT, a serious and life-threatening inherited blood disorder. PRIME designation allows us the opportunity to engage more closely with the EMA and potentially accelerate our ability to provide an innovative new treatment option for patients and families affected by TDBT.”