AstraZeneca and Merck & Co have announced that the European Medicines Agency has granted orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1).
“There is no cure for NF1, a life-long and devastating condition, and current treatment choices for these patients are very limited. The granting of an orphan designation is a positive step forward for children with NF1 and their families,” said Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca.
The potential benefit of selumetinib in NF1 is being explored in the Phase I/II SPRINT trial in paediatric patients with inoperable NF1-related PNs. Select findings were presented recently at the 2018 American Society of Clinical Oncology Annual Meeting in Chicago by the principal investigators at the National Cancer Institute. Full results are expected later in 2018.