Policy & Regulation
US FDA Accepts Regulatory Submission of New Drug Application for Selumetinib in Neurofibromatosis Type 1 and Grants Priority Review
15 November 2019 - - The US Food and Drug Administration has accepted a New Drug Application and granted priority review for the MEK 1/2 inhibitor selumetinib as a potential new medicine for pediatric patients aged three years and older with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, English-Swedish drugmaker AstraZeneca and US-based Merck (NYSE: MRK) said.
This is the first acceptance of a regulatory submission for an oral MEK 1/2 monotherapy for patients with NF1, a rare and incurable genetic condition.
A Prescription Drug User Fee Act (PDUFA) date is set for the second quarter of 2020.
This regulatory submission was based on positive results from the National Cancer Institute Cancer Therapy Evaluation Program -sponsored SPRINT Phase 2 Stratum 1 trial.
An objective response rate was achieved in 66% of pediatric patients with NF1 and symptomatic, inoperable PNs (n=33/50 patients) when treated with selumetinib as a twice-daily oral monotherapy. ORR was defined as the %age of patients with a confirmed complete or partial response of ≥ 20% tumor volume reduction.
Selumetinib was granted US FDA Breakthrough Therapy Designation for this population in April of 2019, US FDA Orphan Drug Designation in February of 2018, EU Orphan Drug Designation by the European Medicines Agency in August 2018, and Swissmedic Orphan Drug Status in December 2018.
AstraZeneca and Merck have a strategic collaboration agreement to co-develop and co-commercialize selumetinib globally.
The SPRINT trial is a US NCI CTEP-sponsored Phase 1/2 trial. The Phase 1 trial was designed to identify the optimal Phase 2 dosing regimen, and the results were published in the New England Journal of Medicine.
Selumetinib is an investigational 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.
Neurofibromatosis Type 1 is an incurable genetic condition that affects one in every 3,000 to 4,000 individuals. 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 30-50% 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 as well as having the potential to transform into malignant peripheral nerve sheath tumors (MPNST).
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.
NF1 also increases a person's risk of developing other cancers, including malignant brain tumors, MPNST and leukemia.
Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.
In July 2017, AstraZeneca and Merck, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co- commercialize certain oncology products, including investigational selumetinib, a MEK inhibitor. Working together, the companies will develop selumetinib in combination with other potential new medicines and as monotherapy.
Independently, the companies will develop selumetinib in combination with their respective PD-L1 and PD-1 medicines.
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