The first drug has been approved in Italy for the treatment of tumors called symptomatic and inoperable plexiform neurofibromas (Pn) in pediatric patients with a rare genetic disease, neurofibromatosis type 1 (Nf1) aged three years or more, after conditional approval in the European Union (EU), which occurred in June 2021.
Selumetinib shrinks the size of inoperable tumors in children, reducing pain and improving quality of life.
NF1 is a debilitating genetic condition which affects one in 3,000 people worldwide and which in Italy is estimated to affect around 20,000 people.
In between 30 and 50% of patients, tumors develop on the nerve sheaths (plexiform neurofibromas) causing problems such as disfigurement, motor dysfunction, pain, airway dysfunction, visual disturbances and bladder or intestinal dysfunction.
The approval of Selumetinib by Aifa is based on the positive results of the Sprint Stratum 1 Phase II study, funded by the Cancer Therapy Evaluation Program (CTEP) of the National Cancer Institute (NCI).
"From today, also in Italy, all pediatric patients with symptomatic inoperable plexiform neurofibromatosis can have access to selumetinib and therefore potentially benefit from this treatment, which can have a positive impact on the quality of life, in particular with a lasting reduction in pain", declares Maria Cristina Diana, of the IRCCS Istituto G.Gaslini, Genoa.
"Selumetinib also shows improvements in terms of muscle strength and joint mobility and, more generally, on tumor-related symptoms", adds Antonella Cacchione, of the Irccs Bambino Gesù Children's Hospital, Rome.
"Our experience in clinical practice confirms these data and we believe that the reduction in hospitalization time thanks to this therapy is also an important parameter to consider."
Reproduction reserved © Copyright ANSA