TIDMAZN
RNS Number : 6762W
AstraZeneca PLC
03 August 2018
03 August 2018 07:00BST
Selumetinib granted orphan designation
in Europe for neurofibromatosis type 1
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known
as MSD outside the US 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.(1,2) 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 tumours on
nerve sheaths that develop in 20-50% of patients, and as they
continue to increase in number and size, cause moderate-to-severe
morbidities such as pain, motor dysfunction and disfigurement.
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."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD 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 MSD 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 I/II SPRINT trial in paediatric patients with inoperable
NF1-related PNs. Select findings were presented recently at the
2018 American Society of Clinical Oncology Annual Meeting in
Chicago by the principal investigators at the National Cancer
Institute. 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. 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 US 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 & Co., Inc., Kenilworth, NJ, US entered a
co-development and co-commercialisation 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 signalling, which can cause cells to grow, divide
and copy themselves in an uncontrolled manner, and may result in
tumour growth. Selumetinib inhibits the MEK enzyme in this pathway,
potentially leading to inhibition of tumour 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% of patients, benign tumours 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 tumours, and leukaemia.
Symptoms begin during early childhood, with varying degrees of
severity, and can reduce life expectancy by up to 15 years.(3)
About the 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
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.
About 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 at
least six new medicines to be launched between 2014 and 2020, and a
broad pipeline of small molecules and biologics in development, we
are committed to advance New Oncology as one of AstraZeneca's five
Growth Platforms focused on lung, ovarian, breast and blood
cancers. In addition to our core capabilities, we actively pursue
innovative partnerships and investments that accelerate the
delivery of our strategy, as illustrated by our investment in
Acerta Pharma in haematology.
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.
About AstraZeneca
AstraZeneca 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 &
Metabolism and Respiratory. AstraZeneca operates in over 100
countries and its innovative medicines are used by millions of
patients worldwide.
For more information, please visit www.astrazeneca.com and
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References
1 NHS Choices. Neurofibromatosis Type 1. Available at
https://www.nhs.uk/conditions/neurofibromatosis-type-1/. Accessed
May 2018.
2 Ghalayani P, et al. Neurofibromatosis Type I (von
Recklinghausen's Disease): A Family Case Report and Literature
Review. Dent Res J. 2012;9(4): 483-488.
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