NF1 is a rare and debilitating genetic condition, where 30-50% of patients with NF1 experience PN tumours growing inside their nerve sheaths

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US FDA approves AstraZeneca’s Koselugo for NF1 in children. (Credit: AstraZeneca.)

AstraZeneca and Merck have obtained the US Food and Drug Administration (FDA) approval for Koselugo (selumetinib) to treat a type of neurofibromatosis in paediatric patients.

FDA has indicated Koselugo for neurofibromatosis type 1 (NF1) in paediatric patients aged two years or more, who have symptomatic, inoperable plexiform neurofibromas (PN).

NF1 is a rare and debilitating genetic condition, where 30-50% of patients with NF1 experience PN tumours growing inside their nerve sheaths, causing clinical issues including pain, motor dysfunction, airway dysfunction, bowel or bladder dysfunction and disfigurement.

AstraZeneca oncology business unit executive vice president Dave Fredrickson 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.”

Koselugo the first regulatory approved twice-daily oral monotherapy for NF1 PN

The FDA approval for Koselugo was based on positive results from the Phase 2 SPRINT Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Pediatric Oncology Branch.

The study was sponsored by the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP), and Koselugo is the first regulatory approved medicine for the treatment of NF1 PN.

The study results showed a 66% overall response rate (ORR) in paediatric patients treated with Koselugo as a twice-daily oral monotherapy. ORR is the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumour volume.

SPRINT trial principal investigator Brigitte C Widemann 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.

MSD Research Laboratories senior vice president and global clinical development head Roy Baynes 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.”