AZ and Merck’s selumetinib drug granted by the FDA

The US Food and Drug Administration has approved of Merck and AstraZeneca’s selumetinib drug, a treatment for pediatric patients who are over the age of two suffering with neurofibromatosis type one (NF1). The therapy is the first to be approved for children with the condition who have tumors in the protective sheaths that cover their nerves.

The approvals based on the positive outcome from the National Cancer Institute Cancer Therapy Evaluation Program sponsored Phase 2 SPRINT Stratum one trial. The trial tested the drug as a twice-daily oral monotherapy in pediatric patients, aged three years plus, with inoperable NF1 plexiform neurofibromas.

The FDA approval provides a priority review voucher to Merck and AstraZeneca, which rewards companies that produce rare disease drugs. It can be used to decrease the review time for a potential drug to 6 months.

Executive Vice President of the Oncology Business Unit, Dave Fredrickson, stated: “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, the Children’s Tumor Foundation, the NF1 patient community and most importantly, the children, parents, and doctors who participated in the SPRINT clinical trial program.”

Head of Global Clinical Development, Senior Vice President and Chief Medical Officer at MSD Research Laboratories, Roy Baynes, stated: “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.”