Selumetinib Granted U.S. Breakthrough Therapy Designation in Neurofibromatosis Type 1


April 1, 2019 5:30 am ET

Designation Based on Phase 2 SPRINT Trial in Pediatric Patients with NF1 Plexiform Neurofibromas

Selumetinib Is a MEK 1/2 Inhibitor Being Co-Developed by AstraZeneca and Merck

KENILWORTH, N.J.–(BUSINESS WIRE)–AstraZeneca and Merck (NYSE: MRK), known as MSD outside the United
States and Canada, today announced that the U.S. Food and Drug
Administration (FDA) has granted Breakthrough Therapy Designation (BTD)
for the investigational MEK 1/2 inhibitor selumetinib.

This designation is for the treatment of pediatric patients aged three
years and older with neurofibromatosis type 1 (NF1) symptomatic and/or
progressive, inoperable plexiform neurofibromas (PN), a rare, incurable
genetic condition.

José Baselga, executive vice president, oncology, AstraZeneca, said,
“Selumetinib shows promise in the treatment of NF1-related plexiform
neurofibromas, a rare and debilitating disease with no approved
medications to date. The Breakthrough Therapy Designation acknowledges
the significant unmet need of these patients and the potential benefit
of selumetinib in this setting.”

Dr. Roy Baynes, senior vice president and head of global clinical
development, chief medical officer, Merck Research Laboratories, said,
“This new designation validates our ongoing development of selumetinib.
As a result of this, selumetinib has the potential to receive expedited
regulatory review and we hope to bring this medicine to patients as soon
as possible.”

The BTD is based on Phase 2 data from the SPRINT trial, evaluating
selumetinib as an oral monotherapy in pediatric patients, aged three
years or older with inoperable NF1-related PN. The results of the trial
were presented by the National Cancer Institute (NCI) at the 2018
American Society of Clinical Oncology Annual Meeting.

BTD is designed to expedite the development and regulatory review of
medicines that are intended to treat a serious condition and that have
shown encouraging early clinical results, which may demonstrate
substantial improvement on a clinically-significant endpoint over
available medicines.

Selumetinib was granted Orphan Drug Designation for the treatment of NF1
by the FDA in February 2018 and the European Medicines Agency in August

About Selumetinib

Selumetinib is an investigational MEK 1/2 inhibitor and potential new
medicine licensed by AstraZeneca from Array BioPharma Inc. in 2003.
AstraZeneca and Merck entered a co-development and co-commercialization
agreement for selumetinib in 2017.

The NF1 gene provides instructions for making a protein called
neurofibromin, which negatively regulates the RAS/MAPK pathway, helping
to control cell growth, differentiation and survival. Mutations in the NF1
gene may result in dysregulations in RAS/RAF/MEK/ERK signaling, which
can cause cells to grow, divide and copy themselves in an uncontrolled
manner, and may result in tumor growth. Selumetinib inhibits the MEK
enzyme in this pathway, potentially leading to inhibition of tumor

Selumetinib is being assessed as a monotherapy and in combination with
other treatments in ongoing trials.

About the SPRINT Trial

The SPRINT trial is a U.S. Cancer Therapy Evaluation Program (CTEP)
National Cancer Institute (NCI)-sponsored Phase 1/2 study. The Phase 1
study was designed to identify the optimal Phase 2 dosing regimen, and
the results were published in the New England Journal of Medicine.

About Neurofibromatosis Type 1 (NF1)

Neurofibromatosis type 1 (NF1) is an incurable genetic condition that
affects one in 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 20-50 percent 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

People with NF1 may experience a number of other 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.

About the AstraZeneca and Merck Strategic Oncology Collaboration

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

Merck’s Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology
medicines to help people with cancer worldwide. At Merck, the potential
to bring new hope to people with cancer drives our purpose and
supporting accessibility to our cancer medicines is our commitment. As
part of our focus on cancer, Merck is committed to exploring the
potential of immuno-oncology with one of the largest development
programs in the industry across more than 30 tumor types. We also
continue to strengthen our portfolio through strategic acquisitions and
are prioritizing the development of several promising oncology
candidates with the potential to improve the treatment of advanced
cancers. For more information about our oncology clinical trials, visit

About Merck

For more than a century, Merck, a leading global biopharmaceutical
company known as MSD outside of the United States and Canada, has been
inventing for life, bringing forward medicines and vaccines for many of
the world’s most challenging diseases. Through our prescription
medicines, vaccines, biologic therapies and animal health products, we
work with customers and operate in more than 140 countries to deliver
innovative health solutions. We also demonstrate our commitment to
increasing access to health care through far-reaching policies, programs
and partnerships. Today, Merck continues to be at the forefront of
research to advance the prevention and treatment of diseases that
threaten people and communities around the world – including cancer,
cardio-metabolic diseases, emerging animal diseases, Alzheimer’s disease
and infectious diseases including HIV and Ebola. For more information,
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Pamela Eisele
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Michael Close
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Teri Loxam
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Michael DeCarbo
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