The European Commission has approved GlaxoSmithKline’s ($GSK) Strimvelis, which uses a virus to deliver gene therapy for ADA-SCID, a rare genetic disorder that causes immunodeficiency. The approval comes barely two months after the European Medicines Agency’s safety and efficacy arm gave the treatment a positive opinion.
ADA-SCID, or severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency, affects about 15 children each year in Europe. A child born with ADA-SCID is unable to fight off everyday infections, and without treatment, will typically not survive past age 2. Previously, the standard of care was a bone marrow transplant, which only works in patients who have a well-matched donor.
Strimvelis works by using a virus to insert a normal copy of the ADA gene into a patient’s bone marrow cells. The gene-corrected cells are then reintroduced to the patient using an intravenous infusion. Some of the cells make it back to the bone marrow, and patients can be expected to make the gene on their own.
GlaxoSmithKline has been working on the treatment with Fondazione Telethon in Rome and Ospedale San Raffaele in Milan. Following the approval, ADA-SCID patients who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele.
The marketing authorization comes after a “data package” from 18 children treated with Strimvelis. All 12 of the patients in the pivotal study survived three years following treatment, and all 18 patients who received the treatment in contribution to the “data package” are alive today.