Selumetinib Granted Orphan Designation in Europe for Neurofibromatosis Type 1


August 3, 2018 5:30 am ET

AstraZeneca and Merck (NYSE:MRK), known as MSD outside the U.S. and
Canada, today announced that the European Medicines Agency (EMA) has
granted orphan designation to 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 newborns
worldwide. The severity of signs and symptoms associated with NF1 can be
highly variable, are often mild-to-moderate and may include skin, nerve
and skeletal manifestations. Plexiform neurofibromas (PNs)
are benign tumors on nerve sheaths that develop in 20-50 percent of
patients and, as they continue to increase in number and size, cause
moderate-to-severe morbidities such as pain, motor dysfunction and

Sean Bohen, executive vice president, global medicines development and
chief medical officer, at AstraZeneca, said, “There is no cure for NF1,
a life-long and devastating condition, and current treatment choices for
these patients are very limited. The granting of an orphan designation
is a positive step forward for children with NF1 and their families.”

Dr. Roy Baynes, senior vice president and head of global clinical
development, chief medical officer, Merck Research Laboratories, said,
“NF1 is a relatively rare disease, but can lead to life-threatening
complications in those affected by it. This underscores the importance
of this collaborative effort between Merck and our partner, AstraZeneca,
to help patients impacted by this debilitating genetic condition.”

The potential benefit of selumetinib in NF1 is being explored in the
Phase 1/2 SPRINT trial in pediatric patients with inoperable NF1-related
PNs. Select findings were presented recently at the 2018 American
Society of Clinical Oncology (ASCO) Annual Meeting in Chicago by the
principal investigators at the National Cancer Institute (NCI). Full
results are expected later in 2018.

Orphan designation is a status assigned to a medicine intended for use
in rare diseases. To be granted orphan status by the EMA, a medicine
must be intended for the treatment, prevention or diagnosis of a disease
that is seriously debilitating/life threatening and has a prevalence of
up to five in 10,000 in the European Union (EU). Additionally, the
intended medicine must aim to provide significant benefit to those
affected by the condition. Orphan designation is conferred following a
positive opinion by the EMA’s Committee for Orphan Medicinal Products.
Selumetinib was granted Orphan Drug Designation (ODD) by the U.S. Food
and Drug Administration (FDA) for the treatment of NF1 in February 2018.

About Selumetinib

Selumetinib is an 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 growth. It is also being explored as a monotherapy
and in combination with other treatments in other ongoing trials.

About neurofibromatosis type 1 (NF1)

NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and
affects approximately one in 3,000 births. 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, benign tumors on the nerve sheaths
(plexiform neurofibromas). These plexiform neurofibromas can cause
morbidities such as pain, motor dysfunction and disfigurement.

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 and
peripheral nerve sheath tumors 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 LYNPARZA® (olaparib), 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.

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

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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