AstraZeneca Submits Clinical Application for Selumetinib Capsules in China Following FDA NDA Filing for NF1

Source | PharmaCube Info

Text | Baihuawen

On November 20, the CDE website showed that AstraZeneca submitted a clinical trial application for Selumetinib capsules in China. Notably, this new drug had just been submitted to the U.S. FDA on November 14 for approval as a treatment for patients aged 3 years and older with neurofibromatosis type 1 (NF1) associated with plexiform neurofibromas who are not candidates for surgery, and it has been granted priority review status by the FDA.

Neurofibromatosis type 1 (NF1) is an incurable genetic disorder with an incidence of approximately 1 in 3,000 to 4,000 live births. It is estimated that 20–50% of individuals with NF1 may develop plexiform neurofibromas, tumors that are often not amenable to complete surgical resection and carry a high risk of recurrence. Patients with NF1 may experience numerous complications, such as learning difficulties, visual impairments, spinal deformities and scoliosis, hypertension, and epilepsy. NF1 also increases the risk of developing other cancers, including malignant brain tumors and leukemia.

Selumetinib is a MEK1/2 inhibitor. MEK is a key protein kinase in the RAS/MAPK signaling pathway, and the pathogenesis of NF1-related tumors is driven by NF1 gene mutations that disrupt this pathway. The drug was granted Breakthrough Therapy designation by the FDA in April 2019 and received Orphan Drug designation in February 2018. Under a licensing agreement, AstraZeneca and MSD are jointly developing and commercializing selumetinib globally.

The Phase II clinical trial, codenamed SPRINT, demonstrated that selumetinib monotherapy achieved an objective response rate (ORR) of 66% in patients with neurofibromatosis type 1 (NF1). ORR was defined as complete tumor regression or a reduction in tumor volume exceeding 20%. Based on these clinical trial results, AstraZeneca/MSD submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date for the second quarter of 2020. If approved, this drug would become the first oral MEK1/2 inhibitor authorized by the FDA for the treatment of NF1.