Koselugo has been designed to treat symptomatic, inoperable nerve tumours in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.
It is the first time a drug for NF1, which is a debilitating genetic condition affecting one in 3,000 individuals worldwide, has been approved in the region.
In 30-50% of people with NF1, tumours develop on the nerve sheaths, called plexiform neurofibromas, and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.
They start developing during early childhood, with varying degrees of severity, and can reduce life expectancy by eight to 15 years.
The approval by the European Commission was based on positive results from a mid-stage trial sponsored by the National Institute of Health's National Cancer Institute Cancer Therapy Evaluation Program.
Koselugo reduced the size of inoperable tumours in 66% of children, reducing pain and improving quality of life.
Koselugo is approved in the US and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable plexiform neurofibromas.
Clinical trials with adult patients, including an alternative age-appropriate formulation for paediatric patients, are scheduled to begin this year, the FTSE 100 group said.