Adnkronos Rome, October 9 (Adnkronos Salute) – "Patients" have been waiting "for a long time" for this treatment that allows them to "be healed." The work is now in the hands of the regions "because they will have to identify the centers designated to administer the therapy. We have 4-5 throughout Italy that are authorized and capable of doing so." In particular, it would be important for "Sardinia and Sicily—the regions" where hemoglobinopathies are "endemic, with approximately 2.600-2.700 patients—to nominate their own centers: this would avoid complicated travel."
This is how Valentino Orlandi, president of United, the National Federation of Patient Associations with Thalassemia or Sickle Cell Anemia, commented on the publication in the Official Journal by the Italian Medicines Agency (AIFA) regarding the reimbursement of exagamglogene autotemcel (exa-cel), the first and only gene editing therapy approved for the treatment of patients with transfusion-dependent beta-thalassemia (TDT) and severe sickle cell anemia (SCD).
The new treatment with Crispr-Cas9 technology (the 'molecular scissors' of gene editing) is indicated for patients with specific characteristics and defined age limits. "This is a kind of discrimination," observes Orlandi. "My work will be ongoing and constant" to ensure timely access for eligible patients. "At our conferences, we've heard testimonials from patients who underwent this therapy in clinical trials, two or three years ago: they developed significant fetal hemoglobin levels, with an energy and strength they hadn't seen before. Therefore," he emphasizes, "it won't be for all patients, it won't be tomorrow, but these patients" for whom the therapy is available "will turn their lives around."