Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1


February 15, 2018 6:30 am ET

AstraZeneca and Merck (NYSE:MRK), known as MSD outside the U.S. and
Canada, today announced that the U.S. Food and Drug Administration (FDA)
has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2
inhibitor, for the treatment of neurofibromatosis type 1 (NF1).

NF1 is an incurable genetic condition that affects one in 3,000 births
with highly-variable symptoms including cutaneous (skin), neurological
(nervous system) and orthopedic (skeletal) manifestations. NF1 can cause
secondary complications including learning difficulties, visual
impairment, pain, disfigurement, twisting and curvature of the spine,
high blood pressure and epilepsy. Plexiform neurofibromas (PNs) are
tumors that arise from nerve fascicles and tend to grow along the length
of the nerve. PNs, a neurological manifestation of NF1,occur in
approximately 20-50 percent of NF1 patients causing pain, motor
dysfunction and disfigurement.

Sean Bohen, executive vice president, global medicines development and
chief medical officer, AstraZeneca, said, “Neurofibromatosis type 1 is a
devastating condition that can lead to life-threatening complications.
There is no known cure for neurofibromatosis and there are limited
treatment options to manage symptoms.”

Dr. Roy Baynes, senior vice president and head of global clinical
development, chief medical officer, Merck Research Laboratories, said,
“This is an important collaborative effort with our colleagues at
AstraZeneca addressing an area of significant unmet medical need to
potentially benefit patients with neurofibromatosis type 1.”

The potential benefit of selumetinib in NF1 is being explored in the
U.S. National Cancer Institute-sponsored phase 1/2 SPRINT trial in
pediatric patients with symptomatic NF1-related PNs. Phase II trial
results are expected later in 2018.

The FDA’s ODD program provides orphan status to medicines that are
defined as those intended for the safe and effective treatment,
diagnosis or prevention of rare diseases or disorders that affect fewer
than 200,000 people in the U.S.

In addition to NF1, selumetinib is being investigated in the phase 3
ASTRA trial of patients who are diagnosed with differentiated thyroid
cancer (DTC) following surgery and treatment with radioactive iodine.
Selumetinib was granted ODD by the US FDA for the adjuvant treatment of
stage 3/4 DTC in 2016. It is also being explored as a monotherapy and in
combination with other treatments in phase 1 trials.


About neurofibromatosis type 1 (NF1)

The NF1 gene provides instructions for making a protein called
Neurofibromin. The disease is associated with many symptoms, including
soft lumps on and under the skin (subcutaneous neurofibromas), skin
pigmentation (cafe au lait spots) and, in 20-50 percent of patients,
tumors on the nerve sheaths (plexiform neurofibromas). These plexiform
neurofibromas can cause morbidities such as pain, motor dysfunction and
disfigurement. Patients 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. People
with NF1 also have an increased risk of developing other cancers,
including malignant brain and peripheral nerve sheath tumors, and
leukaemia. Symptoms begin during early childhood, with varying degrees
of severity, and can reduce life expectancy by up to 15 years.

About selumetinib

Selumetinib, is an investigational MEK 1/2 inhibitor licensed by
AstraZeneca from Array BioPharma Inc. in 2003.

The NF1 gene codes for a protein called Neurofibromin. This protein
negatively regulates the RAS/MAPK pathway, which helps to control cell
growth, differentiation and survival. Mutations in the NF1 gene may
result in dysregulation 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 growth.

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 LYNPARZA, the world’s first PARP
inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for
multiple cancer types. The collaboration is based on increasing evidence
that PARP and MEK inhibitors can be combined with PD-L1/PD-1 inhibitors
for a range of tumor types. Working together, the companies will jointly
develop LYNPARZA and selumetinib in combination with other potential new
medicines and as a monotherapy. Independently, the companies will
develop LYNPARZA and selumetinib in combination with their respective
PD-L1 and PD-1 medicines.

Merck’s Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology
medicines to help people with cancer worldwide. At Merck, helping people
fight cancer is our passion and supporting accessibility to our cancer
medicines is our commitment. Our focus is on pursuing research in
immuno-oncology and we are accelerating every step in the journey – from
lab to clinic – to potentially bring new hope to people with cancer.

As part of our focus on cancer, Merck is committed to exploring the
potential of immuno-oncology with one of the fastest-growing development
programs in the industry. We are currently executing an expansive
research program evaluating our anti-PD-1 therapy across more than 30
tumor types. We also continue to strengthen our immuno-oncology
portfolio through strategic acquisitions and are prioritizing the
development of several promising immunotherapeutic 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, 267-305-3558
Courtney Ronaldo, 908-442-5695
Teri Loxam, 908-740-1986
Michael DeCarbo, 908-740-1807

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