AstraZeneca gets USFDA nod for Koselugo for paediatric patients with neurofibromatosis type 1

Kenilworth: AstraZeneca and MSD Inc. has announced that the US Food and Drug Administration (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 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.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "For the first time, patients and families impacted by this incurable genetic condition have an approved medicine to treat the resulting plexiform neurofibromas. I would like to thank our research partners, the NCI, the Neurofibromatosis Therapeutic Acceleration Program (NTAP), the Children's Tumor Foundation (CTF), the NF1 patient community and, most importantly, the children, parents and doctors who participated in the SPRINT clinical trial programme."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Previously there were no medicines approved for this disease. This approval has the potential to change how symptomatic, inoperable NF1 plexiform neurofibromas are treated and provides new hope for these patients."

Brigitte C. Widemann, M.D., Principal Investigator of the SPRINT trial and Chief, National Cancer Institute Pediatric Oncology Branch, said: "Koselugo has made a difference for many children in this trial. This is an important treatment advance for patients and their families."

"AstraZeneca and MSD are jointly developing and commercialising Koselugo globally and submitted a marketing authorisation application in NF1 PN to the European Medicines Agency in the first quarter of 2020. Further global regulatory submissions are being evaluated", Release stated

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