Aifa has given the green light to the reimbursement of the first drug therapy for the treatment of retinopathy of the premature baby (Rop), a retinal disease that affects 26.9% of premature births and is responsible for 6-18% of childhood blindness. This is ranibizumab which, having demonstrated clinical superiority, in terms of efficacy and safety, compared to laser therapy, the current standard of care, can be prescribed and reimbursed for this new indication from the beginning of the month.
Retinopathy of the premature baby
Retinopathy of the premature baby is a vascular disease of the retina that occurs in premature babies before the thirty-second week of gestation and / or with a birth weight of less than 1.5 kg, and is due to the abnormal development of the blood vessels of the retina, which normally occurs in week 34-36 of gestation. The progression of the disease depends, in fact, on the high levels of VEGF (vascular endothelial growth factor), the growth factor that governs retinal vasculogenesis, which after premature birth, in high levels, can lead to structural abnormalities such as retinal detachment, low vision and blindness.
How the therapy works
Ranibizumab, as an intravitreal anti-VEGF therapy, acting in a pharmacologically selective manner reduces the high level of growth of the vascular endothelium, significantly improving the prognosis of treated patients. Until now, this pathology was treated with laser therapy which works by destroying the eye tissue that contributes to the increase in VEGF. But while effective, this therapy is often associated with irreversible retinal damage such as severe myopia, strabismus, cataracts, amblyopia and glaucoma.
The confirmation of a medical practice
“This approval is of particular importance – comments the Domenico Lepore, Catholic University of the Sacred Heart A. Gemelli Irccs Polyclinic Foundation – since the use of anti-VEGF therapy in retinopathy of the premature baby had already established itself for some time in our medical practice. Furthermore, as reported in the latest publication in the Lancet journal – continues Lepore – the child treated with ranibizumab not only has less myopia and a more physiologically developed retina that does not report scars, but above all face-motor perception tests have shown in these little ones patients greater vision and better performance than those treated with laser “.
Lo studio Rainbow
The results of the Rainbow study, on which the Ema approval and reimbursement just received by Aifa are based, show a higher benefit / risk profile of ranibizumab compared to laser therapy, considered to date the therapeutic gold standard. In particular, greater efficacy in terms of treatment success measured by the absence of active disease and unfavorable structural outcomes in both eyes, higher than that observed in surgically treated patients (80% vs 66.2%); less damage in the ocular anatomy compared to laser therapy (1.4% vs 10.1%); long-term safety profile in preterm infants confirmed at 2 years. Additionally, 78% of pharmacologically treated patients received a single injection per eye.