US FDA accepts regulatory submission for selumetinib in neurofibromatosis type 1 and grants priority review

AstraZeneca and Merck’s selumetinib would become the first medicine indicated for the treatment of pediatric patients with NF1 plexiform neurofibromas if approved

AstraZeneca and Merck & Co., Inc., Kenilworth, N.J., US (Merck: known as MSD outside the US and Canada) today announced that the US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) and granted Priority Review for selumetinib as a potential new medicine for pediatric patients aged three years or older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PNs).

This is the first acceptance of a regulatory submission for a MEK 1/2 inhibitor as an oral monotherapy for pediatric patients with NF1 PNs. A Prescription Drug User Fee Act (PDUFA) date is set for the second quarter of 2020.

The regulatory submission was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored SPRINT Phase II Stratum 1 trial, which showed that 66% of pediatric patients with NF1 and symptomatic, inoperable PNs (n=33/50 patients) achieved an objective response rate (ORR) when treated with selumetinib as a twice-daily oral monotherapy. ORR was defined as the percentage of patients with a confirmed complete or partial response of ≥ 20% tumor volume reduction.

Selumetinib, a MEK 1/2 inhibitor was granted US FDA Breakthrough Therapy Designation in April 2019 and Orphan Drug Designation in February 2018. AstraZeneca and Merck are jointly developing and commercializing selumetinib globally under a license agreement.

NOTES TO EDITORS

About SPRINT
SPRINT is a US Cancer Therapy Evaluation Program (CTEP) NCI-sponsored Phase I/II trial. The Phase I trial was designed to identify the optimal Phase II dosing regimen, and the results were published in The New England Journal of Medicine.

About Selumetinib   
Selumetinib is a MEK 1/2 inhibitor. It is designed to inhibit the MEK enzyme in the RAS/MAPK pathway, a cell-signaling pathway, associated with cancer cell growth and proliferation in a number of different tumor types.

About NF1 with Plexiform Neurofibromas (PN)
Neurofibromatosis type 1 (NF1) is an incurable genetic condition that affects one in every 3,000 to 4,000 individuals in the U.S. It is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas), skin pigmentation (so-called ‘cafe au lait’ spots) and, in 20-50% of patients, tumors develop on the nerve sheaths (plexiform neurofibromas). These plexiform neurofibromas can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement.

People with NF1 may experience a number of complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.

About the AstraZeneca and Merck Strategic Oncology Collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialize LYNPARZA, the world’s first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop LYNPARZA and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop LYNPARZA and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

About AstraZeneca in Oncology
AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in hematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumor Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalized combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

About AstraZeneca
AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism and Respiratory. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information, please visit www.astrazeneca-us.com and follow us on Twitter @AstraZenecaUS.


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US-31292 Last Updated 11/19