AstraZeneca PLC (LON:AZN) and Merck have had a US new drug application accepted and been granted a priority review for their treatment for neurofibromas or non-cancerous tumours in children.
If approved, the drug, selumetinib, would become the first medicine indicated for the treatment of paediatric patients with NF1 plexiform neurofibromas.
NF1 is a rare and incurable genetic condition that causes skin abnormalities and non-cancerous tumours to grow thoughout the body.
The application is for the use of selumetinib in children aged three years and older.
AstraZeneca said is the first acceptance of a regulatory submission for an oral monotherapy for the treatment of NF1, with A Prescription Drug User Fee Act (PDUFA) date set for the second quarter of 2020.
Selumetinib was granted US FDA Breakthrough Therapy Designation in April 2019, with the NDA submission based on positive results from the National Cancer Institute-sponsored SPRINT Phase II Stratum 1 trial.