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.
NOTES TO EDITORS
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
www.merck.com/clinicaltrials.
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,
visit www.merck.com and connect with us on
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