Aifa, the Italian Medicines Agency, has recently approved the first drug for the treatment of rare tumors in children. This groundbreaking medication is specifically designed to target symptomatic and inoperable neurofibromas, also known as Pn tumors, in pediatric patients with a rare genetic disease called neurofibromatosis type 1 (Nf1) who are three years old or older. The conditional approval from the European Union (EU) was granted in June 2021.
The newly approved drug, Selumetinib, has shown promising results in reducing the size of inoperable tumors in children. It not only helps to alleviate pain but also improves the overall quality of life for these young patients.
Neurofibromatosis type 1 (Nf1) is a debilitating genetic condition that affects approximately one person out of every 3,000 worldwide. In Italy alone, it is estimated that around 20,000 people are affected by this condition. Between 30% and 50% of patients with Nf1 develop tumors on their nerve sheaths, known as neurofibromas plexiforms. These tumors can cause a variety of issues such as deformities, motor dysfunctions, pain, airway problems, visual impairments, and bladder or bowel dysfunctions.
The approval of Selumetinib by Aifa is based on the positive results from the Phase II Sprint Stratum 1 study. This study was funded by the Cancer Therapy Evaluation Program (Ctep) of the National Cancer Institute (Nci). “Starting today, all pediatric patients with symptomatic and inoperable neurofibromatosis plexiforme in Italy can have access to Selumetinib and potentially benefit from this treatment, which can have a positive impact on their quality of life, particularly through long-lasting pain reduction,” says Maria Cristina Diana from IRCCS Institute G. Gaslini in Genoa. Antonella Cacchione from IRCCS Bambino Gesù Children’s Hospital in Rome adds, “Selumetinib has also shown improvements in terms of muscle strength, joint mobility, and overall tumor-related symptoms.”
The approval of this drug marks a significant milestone in the treatment of rare tumors in children. The positive results from clinical trials and the experiences of healthcare professionals indicate that Selumetinib can not only reduce tumor size but also improve muscle strength, joint mobility, and overall well-being. Additionally, the reduction in hospitalization time due to this therapy is an important consideration.
The availability of Selumetinib in Italy provides hope for pediatric patients suffering from neurofibromatosis type 1 and its associated tumors. It offers them a potential lifeline, improving their quality of life and providing them with a chance for a brighter future. The approval of this medication showcases the dedication of medical researchers and regulatory agencies to finding effective treatments for rare diseases and ensuring that patients have access to them. With continued advancements in medical research and drug development, the future looks promising for children with rare tumors.