The gene editing therapy “exagamglogene autotemcel” (exa-cel), the first therapy of its kind indicated for the treatment of patients with transfusion-dependent beta-thalassemia and severe sickle cell disease , is officially covered by the National Health Service.
Indeed, the Italian Medicines Agency's decision establishing its reimbursement was published yesterday in the Official Journal.
"Genome editing based on CRISPR/Cas9 technology represents a fundamental turning point for people affected by genetic blood diseases , such as sickle cell anemia and transfusion-dependent beta-thalassemia," explains Franco Locatelli, professor of Pediatrics at the Catholic University of the Sacred Heart in Rome and director of the Department of Pediatric Hematology and Oncology at the Bambino Gesù Children's Hospital, in a statement. "Clinical studies have shown very positive results, and today we are pleased that this innovation is also available in Italy for patients eligible for treatment," added Locatelli, principal investigator of two of the studies that led to the approval of the therapy.
THERAPY – Exa-cel is a therapy in which the patient's hematopoietic stem and progenitor cells are modified to induce the production of high levels of fetal hemoglobin. In trials, it has been shown to reduce or eliminate vaso-occlusive crises in patients with severe sickle cell disease and the need for transfusions in patients with transfusion-dependent beta-thalassemia.
"Thanks to the fruitful collaboration with Italian health authorities, an agreement has been reached that recognizes the value of an innovative, one-time therapy capable of generating a profound impact not only for patients, but also for their families and the entire country," said Federico Viganò, country manager for Italy and Greece at Vertex Pharmaceuticals.
(Unioneonline/vl)
