AstraZeneca and Merck’s Selumetinib Would
Become the First Medicine Indicated for the Treatment of Certain
Pediatric Patients with NF1 Plexiform Neurofibromas if
Approved
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 accepted a New Drug Application (NDA)
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 (NF1) and symptomatic,
inoperable plexiform neurofibromas (PNs).
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 (NCI) Cancer Therapy Evaluation
Program (CTEP)-sponsored SPRINT Phase 2 Stratum 1 trial. An
objective response rate (ORR) 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 percentage of patients with a
confirmed complete or partial response of ≥ 20% tumor volume
reduction.
Selumetinib was granted U.S. FDA Breakthrough Therapy
Designation for this population in April of 2019, U.S. 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.
About SPRINT The SPRINT trial is a U.S. 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.
About Selumetinib 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.
About Neurofibromatosis Type 1 (NF1) NF1 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.
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 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
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 Twitter, Facebook, Instagram, YouTube and LinkedIn.
Forward-Looking Statement of Merck & Co., Inc.,
Kenilworth, N.J., USA This news release of Merck & Co.,
Inc., Kenilworth, N.J., USA (the “company”) includes
“forward-looking statements” within the meaning of the safe harbor
provisions of the U.S. Private Securities Litigation Reform Act of
1995. These statements are based upon the current beliefs and
expectations of the company’s management and are subject to
significant risks and uncertainties. There can be no guarantees
with respect to pipeline products that the products will receive
the necessary regulatory approvals or that they will prove to be
commercially successful. If underlying assumptions prove inaccurate
or risks or uncertainties materialize, actual results may differ
materially from those set forth in the forward-looking
statements.
Risks and uncertainties include but are not limited to, general
industry conditions and competition; general economic factors,
including interest rate and currency exchange rate fluctuations;
the impact of pharmaceutical industry regulation and health care
legislation in the United States and internationally; global trends
toward health care cost containment; technological advances, new
products and patents attained by competitors; challenges inherent
in new product development, including obtaining regulatory
approval; the company’s ability to accurately predict future market
conditions; manufacturing difficulties or delays; financial
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future events or otherwise. Additional factors that could cause
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Annual Report on Form 10-K and the company’s other filings with the
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Internet site (www.sec.gov).
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