FDA approves Koselugo to treat paediatric patients two years of age and older with neurofibromatosis type 1 (NF1). – AstraZeneca + Merck Inc.

AstraZeneca and Merck Inc.announced that the FDA has approved the kinase inhibitor Koselugo (selumetinib) for the treatment of paediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

The approval by the FDA was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored Phase II SPRINT Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Pediatric Oncology Branch. This is the first regulatory approval anywhere in the world of a medicine for the treatment of NF1 PN.

NF1 is a rare and debilitating genetic condition. Some 30-50% of patients with NF1 experience PN – tumours growing inside their nerve sheaths. These PN can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement.

Results showed an overall response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with Koselugo as a twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumour volume.