TIDMAZN
RNS Number : 3486T
AstraZeneca PLC
14 November 2019
14 November 2019 07:00 GMT
US FDA accepts regulatory submission for selumetinib
in neurofibromatosis type 1 and grants Priority Review
AstraZeneca and MSD's selumetinib would become the first
medicine indicated for the treatment of paediatric patients with
NF1 plexiform neurofibromas if approved
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 accepted a New
Drug Application (NDA) and granted Priority Review for selumetinib
as a potential new medicine for paediatric 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 monotherapy for the treatment of NF1, a rare and incurable
genetic condition. A Prescription Drug User Fee Act (PDUFA) date is
set for the second quarter of 2020.
The regulatory submission was based on positive results from the
National Cancer Institute (NCI) Cancer Therapy Evaluation Program
(CTEP)-sponsored SPRINT Phase II Stratum 1 trial. An Objective
Response Rate (ORR) was achieved in 66% of paediatric 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% tumour volume reduction.
Selumetinib, a MEK 1/2 inhibitor was granted US FDA Breakthrough
Therapy Designation in April 2019, Orphan Drug Designation in
February 2018, EU Orphan Designation in August 2018 and Swissmedic
Orphan Drug Status in December 2018. AstraZeneca and MSD are
jointly developing and commercialising selumetinib globally under a
license agreement.
About SPRINT
SPRINT is a US NCI CTEP-sponsored Phase I/II trial. The Phase I
trial was designed to identify the optimal Phase II dosing regimen,
and the results were published in The New England Journal of
Medicine.(1)
About selumetinib
Selumetinib is a MEK 1/2 inhibitor. It is designed to inhibit
the MEK enzyme in the RAS/MAPK pathway, a cell-signalling pathway,
associated with cancer cell growth and proliferation in a number of
different tumour types.
About NF1
NF1 is an incurable genetic condition that affects one in every
3,000 to 4,000 individuals.(2,3) 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, tumours develop on the nerve sheaths
(plexiform neurofibromas).(1) 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 tumours (MPNST).(1)
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 tumours, MPNST and leukaemia. Symptoms begin during early
childhood, with varying degrees of severity, and can reduce life
expectancy by up to 15 years.(4)
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 Oncology as one of AstraZeneca's four
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 and
Metabolism, and Respiratory. AstraZeneca operates in over 100
countries and its innovative medicines are used by millions of
patients worldwide. Please visit astrazeneca.com and follow the
Company on Twitter @AstraZeneca.
Media Relations
Gonzalo Viña +44 203 749 5916
Rob Skelding Oncology +44 203 749 5821
Rebecca Einhorn Oncology +1 301 518 4122
Matt Kent BioPharmaceuticals +44 203 749 5906
Jennifer Hursit Other +44 203 749 5762
Christina Malmberg Hägerstrand Sweden +46 8 552 53 106
Michele Meixell US +1 302 885 2677
Investor Relations
Thomas Kudsk Larsen +44 203 749 5712
Henry Wheeler Oncology +44 203 749 5797
Christer Gruvris BioPharmaceuticals (CV, Metabolism) +44 203 749 5711
Nick Stone BioPharmaceuticals (Renal), ESG +44 203 749 5716
BioPharmaceuticals (Respiratory),
Josie Afolabi other medicines +44 203 749 5631
Craig Marks Finance, fixed income +44 7881 615 764
Jennifer Kretzmann Corporate access, retail investors +44 203 749 5824
US toll-free +1 866 381 72 77
References
1. Dombi E, et al. Activity of Selumetinib in Neurofibromatosis
Type 1-Related Plexiform Neurofibromas. N Engl J Med.
2016;375:2550-2560.
2. National Institute of Neurological Disorders and Stroke.
Neurofibromatosis Fact Sheet. "What is NF1?" Available at:
https://www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/neurofibromatosis-fact-sheet
#3162_2 Accessed October 2019.
3. ASCO Cancer.Net. Neurofibromatosis Type 1. Available at
https://www.cancer.net/cancer-types/neurofibromatosis-type-1
Accessed June 2019.
4. Evans DGR, et al. Reduced Life Expectancy Seen in Hereditary
Diseases Which Predispose to Early-Onset Tumors. Appl Clin Genet.
2013;6:53-61.
Adrian Kemp
Company Secretary
AstraZeneca PLC
This information is provided by RNS, the news service of the
London Stock Exchange. RNS is approved by the Financial Conduct
Authority to act as a Primary Information Provider in the United
Kingdom. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
END
REAFXLLFKFFEFBB
(END) Dow Jones Newswires
November 14, 2019 02:01 ET (07:01 GMT)
Astrazeneca (LSE:AZN)
Historical Stock Chart
From Mar 2024 to Apr 2024
Astrazeneca (LSE:AZN)
Historical Stock Chart
From Apr 2023 to Apr 2024
