The European Commission (EC) has granted AstraZeneca (AZ) and MSD’s Koselugo (selumetinib) a conditional approved for the treatment of children with symptomatic, inoperable plexiform neurofibromas (PN) and neurofibromatosis type 1 (NF1).

The approval is based on results from the SPRINT Stratum 1 Phase II trial, which was designed to evaluate the objective response rate (ORR) and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with Koselugo monotherapy.

This study showed Koselugo reduced the size of inoperable tumours in children, and also reduced pain and improved quality of life for individuals with the rare genetic disease.

Koselugo also demonstrated an ORR of 66% in paediatric patients with NF1 PN when treated with the drug as twice-daily oral monotherapy in this study.

"As the first medicine approved in the EU for patients with neurofibromatosis type 1, Koselugo has the potential to transform the way plexiform neurofibromas are managed and treated. The SPRINT data showed that Koselugo not only shrank tumours in some children, but also reduced pain and improved their quality of life,” said Dave Fredrickson, executive vice president, oncology business unit, AZ.

“This significant milestone was made possible thanks to our research partners, the National Cancer Institute, the Neurofibromatosis Therapeutic Acceleration Program, the Children's Tumor Foundation, the patient community and every child, parent and doctor involved in the clinical trial,” he added.

NF1 is a rare genetic condition that affects one in 3,000 individuals globally – in 30-50% of people with NF1, tumours develop in the nerve sheaths which can cause clinical issues including disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.