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AstraZeneca: Koselugo (selumetinib) approved in US for paediatric patients with neurofibromatosis type 1 plexiform neurofib...

First medicine approved to treat this rare and debilitating genetic

AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck &
Co., Inc. inside the US and Canada) today announced that the US Food
and Drug Administration (FDA) has approved the kinase inhibitor
Koselugo (selumetinib) for the treatment of paediatric patients two
years of age and older with neurofibromatosis type 1 (NF1) who have
symptomatic, inoperable plexiform neurofibromas (PN).[1]

The approval by the FDA was based on positive results from the
National Cancer Institute (NCI) Cancer Therapy Evaluation Program
(CTEP)-sponsored Phase II SPRINT Stratum 1 trial coordinated by the
NCI's Center for Cancer Research, Pediatric Oncology Branch. This is
the first regulatory approval anywhere in the world of a medicine for
the treatment of NF1 PN.[1]

NF1 is a rare and debilitating genetic condition.[2] Some 30-50% of
patients with NF1 experience PN - tumours growing inside their nerve
sheaths. These PN can cause clinical issues such as pain, motor
dysfunction, airway dysfunction, bowel/bladder dysfunction and

Results showed an overall response rate (ORR) of 66% (33 of 50
patients, confirmed partial response) in paediatric patients with NF1
PN when treated with Koselugo as a twice-daily oral monotherapy. ORR
is defined as the percentage of patients with confirmed complete or
partial response of at least 20% reduction in tumour volume.

Dave Fredrickson, Executive Vice President, Oncology Business Unit,
said: "For the first time, patients and families impacted by this
incurable genetic condition have an approved medicine to treat the
resulting plexiform neurofibromas. I would like to thank our research
partners, the NCI, the Neurofibromatosis Therapeutic Acceleration
Program (NTAP), the Children's Tumor Foundation (CTF), the NF1
patient community and, most importantly, the children, parents and
doctors who participated in the SPRINT clinical trial programme."

Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD Research Laboratories, said:
"Previously there were no medicines approved for this disease. This
approval has the potential to change how symptomatic, inoperable NF1
plexiform neurofibromas are treated and provides new hope for these

Brigitte C. Widemann, M.D., Principal Investigator of the SPRINT trial
and Chief, National Cancer Institute Pediatric Oncology Branch, said:
"Koselugo has made a difference for many children in this trial. This
is an important treatment advance for patients and their families."

AstraZeneca and MSD are jointly developing and commercialising
Koselugo globally and submitted a marketing authorisation application
in NF1 PN to the European Medicines Agency in the first quarter of
2020. Further global regulatory submissions are being evaluated.

Priority Review Voucher

AstraZeneca has received a Priority Review Voucher (PRV) under the
Rare Pediatric Disease Designation Program
intended to encourage development of new medicines for rare
paediatric diseases. A PRV entitles the holder to FDA Priority Review
of a single New Drug Application or Biologics License Application,
which reduces the target review time and has led to an expedited

Financial considerations

In accordance with the existing collaboration agreement between Merck
and AstraZeneca, following approval and upcoming launch, AstraZeneca
will book all monotherapy Product Sales of Koselugo; half of gross
profits will be due to Merck and will be recorded under Cost of
Sales. Any potential future sales-related milestone payments will be
recorded under Collaboration Revenue. AstraZeneca will supply


NF1 is a debilitating genetic condition that affects one in every
3,000 to 4,000 individuals.[4] 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) and skin pigmentation (so-called `café au lait'
spots)[2 ]and, in 30-50% of patients, tumours develop on the nerve
sheaths (plexiform neurofibromas). These plexiform neurofibromas can
cause clinical issues such as disfigurement, motor dysfunction, pain,
airway dysfunction, visual impairment, and bladder/bowel dysfunction.
PN begin during early childhood, with varying degrees of severity,
and can reduce life expectancy by up to 15 years.[2,4]


The SPRINT Phase I/II trial was designed to evaluate the objective
response rate and impact on patient-reported and functional outcomes
in paediatric patients with NF1-related inoperable PNs treated with
Koselugo monotherapy.[1] Results were published in The New England
Journal of Medicine
([5] This trial
sponsored by NCI CTEP was conducted under a Cooperative Research and
Development Agreement between NCI and AstraZeneca with additional
support from NTAP.


Koselugo (selumetinib) is inhibitor of mitogen-activated protein
kinase kinases 1 and 2 (MEK1/2). MEK1/2 proteins are upstream
regulators of the extracellular signal-related kinase (ERK) pathway.
Both MEK and ERK are critical components of the RAS-regulated
RAF-MEK-ERK pathway, which is often activated in different types of

Koselugo was granted US FDA Breakthrough Therapy Designation
in April 2019, Rare Pediatric Disease Designation in December 2019,
Orphan Drug Designation in February 2018, EU orphan designation
in August 2018 and Swissmedic Orphan Drug Status in December 2018 for
the treatment of paediatric patients with NF1 PN.

AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US,
known as MSD outside the United States and Canada, announced a global
strategic oncology collaboration to co-develop and co-commercialise
Lynparza, the world's first PARP inhibitor, and Koselugo, a MEK
inhibitor, for multiple cancer types. Working together, the companies
will develop Lynparza and Koselugo in combination with other
potential new medicines and as monotherapies. Independently, the
companies will develop Lynparza and Koselugo in combination with
their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a
quickly growing portfolio of new medicines that has the potential to
transform patients' lives and the Company's future. With six new
medicines launched between 2014 and 2020, and a broad pipeline of
small molecules and biologics in development, the Company is
committed to advance oncology as a key growth driver for AstraZeneca
focused on lung, ovarian, breast and blood cancers. In addition to
AstraZeneca's main capabilities, the Company is actively pursuing
innovative partnerships and investments that accelerate the delivery
of our strategy, as illustrated by the investment in Acerta Pharma in

By harnessing the power of four scientific platforms -
Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response
and Antibody Drug Conjugates - and by championing the development of
personalised combinations, AstraZeneca has the vision to redefine
cancer treatment and one day eliminate cancer as a cause of death.


AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led
biopharmaceutical company that focuses on the discovery, development
and commercialisation of prescription medicines, primarily for the
treatment of diseases in three therapy areas - Oncology,
Cardiovascular, Renal and Metabolism, and Respiratory. Based in
Cambridge, UK, AstraZeneca operates in over 100 countries and its
innovative medicines are used by millions of patients worldwide.
Please visit ( and follow
the Company on Twitter @AstraZeneca


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1. Koselugo (selumetinib) [prescribing information]. Wilmington, DE:
AstraZeneca Pharmaceuticals LP; 2020.

2. National Institute of Neurological Disorders and Stroke.
Neurofibromatosis Fact Sheet. "What is NF1?". Available at:
#3162_2 Accessed February 2020.

3. Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in
neurofibromatosis type 1-related plexiform neurofibromas. N Engl J
Med. 2016;375:2550-2560. DOI: 10.1056/NEJMoa1605943.

4. Rasmussen SA, Yang Q, Friedman JM. Mortality in neurofibromatosis
1: an analysis using U.S. death certificates. Am J Hum Genet.

5. Gross A, Wolters P, Dombi E, et al. Selumetinib in Children with
Inoperable Plexiform Neurofibromas. N Engl J Med. 2020;382: DOI:


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