Orchard's gene therapy, OTL-300, acquired from GlaxoSmithKline, was awarded priority drug eligibility by the European Union

Orchard Therapeutics is a UK biotech company dedicated to changing the lives of people with rare diseases through innovative gene therapies. Recently, the company announced that the European Medicines Agency (EMA) has granted OTL-300 Priority Drug Qualification (PRIME). OTL-300 is an experimental in-body lyviral gene therapy developed for the treatment of transfusion-dependent β thylamic anemia (TDBT), a disease called the HBB gene (β beadin). Rare hereditary blood disease caused by one of the more than 200 mutations in the gene, and the most severe type of β thremia, the only treatment currently is heterogenetic hematopoietic stem cell transplantation, but there is a very high transplant-related morbidity and mortality rate.
PRIME is a rapid approval program launched by EMA in March 2016, similar to the FDA's Breakthrough Therapy Identification (BTD) program, which aims to accelerate the review process of key drugs in the field of pharmaceutical shortages, and the finalists for PRIME's experimental drugs will receive strong support from EMA in clinical trials and drug development to accelerate the development and approval of truly innovative drugs to meet the medical needs of promising new drugs.
EMA awarded OTL-300 PRIME based on data assessing preclinical and early clinical projects for the gene therapy to treat TDBT patients, including data collected from 9 patients in 10 patients in an ongoing proof-of-concept study. As of September 2018, OTL-300 has been evaluated in a total of 9 patients. As of April 2018, the frequency of blood transfusions and the need for blood transfusions were significantly reduced in 5 out of 7 patients who followed at least 12 months. In addition, 3 out of 4 pediatric patients had no blood transfusions within about one month after treatment, 2 out of 3 adult patients had reduced blood transfusion requirements, and 1 had no blood transfusions within 9 months of follow-up. All patients survived. Safety data from the nine patients treated in the study showed good general tolerance for OTL-300.Orchard's pipeline asset
OTL-300 was acquired in April from GlaxoSmithKline (GSK), a pioneering collaboration between GSK and San Rafael Hospital and the Telethon Foundation The gene therapy Strimvelis, the world's first approved in-body gene therapy product, was approved by the European Union in April 2016 to treat children with adenosine desinease deficiency severe combined immunodeficiency (ADA-SCID).
addition to Strimvelis and OTL-300, the autologic gene therapy program in Orchard's pipeline includes products for primary immunodeficiency and neuro-metabolic disorders. (Bio Valley)