Posted by Michael Wonder on 10 Apr 2020
FDA approves first therapy for children with debilitating and disfiguring rare disease
10 April 2020 - Today, the U.S. FDA approved Koselugo (selumetinib) for the treatment of paediatric patients, 2 years of age and older, with neurofibromatosis type 1, a genetic disorder of the nervous system causing tumours to grow on nerves.
Koselugo is the first drug approved by the FDA to treat this debilitating, progressive and often disfiguring rare disease that typically begins early in life.
The FDA approved Koselugo based on a clinical trial conducted by the National Cancer Institute of paediatric patients who had neurofibromatosis type 1 and inoperable plexiform neurofibromas (defined as a plexiform neurofibroma that could not be completely removed without risk for substantial morbidity to the patient).
