EMA Grants PRIME Designation to Orchard Therapeutics’ Beta-Thalassemia Gene Therapy

Rare Daily Staff

The European Medicines Agency granted Orchard Therapeutics Priority Medicines (PRIME) designation to OTL-300, an experimental gene therapy for the treatment of transfusion-dependent beta-thalassemia, the most severe form of the rare inherited blood disorder.

Transfusion-dependent beta-thalassemia (TDBT) is caused by one of more than 200 mutations in the HBB gene. TDBT is characterized by failure to grow and gain weight, infection, and life-threatening anemia, which occur within the first two years of life. The only curative approach is allogeneic hematopoietic stem cell transplantation, which carries significant risks. Most patients with TDBT undergo chronic blood transfusions due to the severity of their symptoms and, as a result, often develop side effects.

OLT-300 is an experiment autologous ex vivo lentiviral gene therapy. Orchard acquired OTL-300 from GSK in April 2018. It originated from a collaboration between GSK, the Hospital San Raffaele, and the Telethon Foundation.

PRIME designation for OTL-300 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, five patients experienced significant reductions in transfusion frequency and volume requirements. Additionally, three of the four pediatric patients were transfusion-free since approximately one month after treatment. 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. OTL-300 was generally well-tolerated.

The PRIME program is designed 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 optimize 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.

“We are pleased that the EMA has granted PRIME designation for OTL-300 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,” said Mark Rothera, president and CEO of Orchard. “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.”

October 8, 2018
Photo: Mark Rothera, president and CEO of Orchard