TIDMAZN
RNS Number : 9694E
AstraZeneca PLC
15 February 2018
15 February 2018 07:00 GMT
SELUMETINIB GRANTED ORPHAN DRUG DESIGNATION BY THE US FDA FOR
NEUROFIBROMATOSIS TYPE 1
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (known
as MSD outside the US and Canada) today announced that the US 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,([i]) with highly-variable symptoms, including cutaneous
(skin), neurological (nervous system) and orthopaedic (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 a neurological
manifestation of NF1 and arise from nerve fascicles that tend to
grow along the length of the nerve. PNs occur in approximately
20-50% of NF1 patients causing pain, motor dysfunction and
disfigurement.([ii])
Sean Bohen, Executive Vice President, Global Medicines
Development and Chief Medical Officer, at 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."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, at MSD Research Laboratories,
said: "We're looking forward to working with our colleagues at
AstraZeneca to develop selumetinib and understand how it may
benefit patients with NF1."
The potential benefit of selumetinib in NF1 is being explored in
the US National Cancer Institute-sponsored Phase I/II SPRINT trial
in paediatric patients with symptomatic NF1-related PNs. Phase II
trial results are expected later in 2018.
The FDA's ODD programme 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 US.
In addition to NF1, selumetinib is being investigated in the
Phase III 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 III/IV DTC in 2016. It is also
being explored as a monotherapy and in combination with other
treatments in Phase I trials.
About neurofibromatosis type 1 (NF1)
NF1 is caused by a spontaneous or inherited mutation in the NF1
gene. 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,
tumours 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 tumours, and leukaemia. Symptoms begin
during early childhood, with varying degrees of severity, and can
reduce life expectancy by up to 15 years.([iii])
About selumetinib
Selumetinib is an investigational MEK 1/2 inhibitor licensed by
AstraZeneca from Array BioPharma Inc. in 2003.
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.
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 (olaparib), 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 tumour types. Working
together, the companies will 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.
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 & Metabolic
Diseases and Respiratory. The Company also is selectively active in
the areas of autoimmunity, neuroscience and infection. 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
follow us on Twitter @AstraZeneca.
Media Relations
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Adrian Kemp
Company Secretary, AstraZeneca PLC
[i] 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.
[ii] Dombi E, et al. Activity of Selumetinib in
Neurofibromatosis Type 1-Related Plexiform Neurofibromas. N Engl J
Med. 2016; 375:2550-2560.
[iii] Evans DGR, et al. Reduced Life Expectancy Seen in
Hereditary Diseases Which Predispose to Early-Onset Tumors. Appl
Clin Genet. 2013; 6:53-61.
This information is provided by RNS
The company news service from the London Stock Exchange
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