TIDMAZN
RNS Number : 4964R
AstraZeneca PLC
30 June 2020
30 June 2020 07:00 BST
Selumetinib granted orphan drug designation
in Japan for neurofibromatosis type 1
Designation follows recent US approval
with additional regulatory submissions underway
Phase II SPRINT trial showed selumetinib reduced tumour
volume in paediatric patients with NF1 plexiform
neurofibromas
AstraZeneca today announced that selumetinib has been granted
orphan drug designation (ODD) in Japan for the treatment of
neurofibromatosis type 1 (NF1), a rare and debilitating genetic
disease.(1)
Selumetinib is co-developed and co-commercialised with MSD Inc.,
Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside
the US and Canada).
Some 30-50% of patients with NF1 experience plexiform
neurofibromas (PN) - tumours growing along their nerve sheaths.(2)
These PN can cause clinical issues such as disfigurement, motor
dysfunction, pain, airway dysfunction, visual impairment and
bowel/bladder dysfunction.(3)
The Japanese Ministry of Health, Labour and Welfare grants ODD
to medicines intended for the treatment of diseases that affect
fewer than 50,000 patients in Japan and for which there is a high
unmet medical need.
José Baselga, Executive Vice President, Oncology R&D, said:
"Neurofibromatosis type 1 can have a devastating impact on children
and new medicines are urgently needed to help treat the resulting
plexiform neurofibromas and associated clinical issues. Current
options in most countries are limited and this designation is a
significant step forward in bringing the first medicine for NF1 to
paediatric patients in Japan."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, MSD Research Laboratories,
said: "Plexiform neurofibromas are one of the key manifestations of
NF1 and can lead to pain and disfigurement. In the SPRINT trial,
selumetinib was shown to reduce the size of these tumours in
children. We are hopeful that we will be able to bring this
treatment to this underserved paediatric patient community in
Japan."
The National Cancer Institute (NCI) Cancer Therapy Evaluation
Program (CTEP)-sponsored Phase I/II SPRINT Stratum 1 trial showed
an overall response rate (ORR) of 66% (33 of 50 patients, confirmed
partial response) in paediatric patients with NF1 PN when treated
with selumetinib 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.
AstraZeneca and MSD are jointly developing and commercialising
selumetinib which was approved in the US in April 2020 under the
medicine name Koselugo for the treatment of paediatric patients two
years and older with NF1 and symptomatic, inoperable PN. A
marketing authorisation application in NF1 PN was accepted for
review by the European Medicines Agency earlier in the year and
further global regulatory submissions are underway.
NF1
NF1 is a debilitating genetic disease that affects one in every
3,000 to 4,000 individuals.(1) 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)(1) and, in 30-50% of patients, tumours develop on the nerve
sheaths (plexiform neurofibromas).(2) These plexiform neurofibromas
can cause clinical issues such as disfigurement, motor dysfunction,
pain, airway dysfunction, visual impairment, and bladder/bowel
dysfunction.(3) PN begin during early childhood, with varying
degrees of severity, and can reduce life expectancy by 8 to 15
years.(1,4,5)
SPRINT
The SPRINT Stratum 1 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 selumetinib monotherapy.(6) Results
were published in The New England Journal of Medicine .(7) This
trial sponsored by NCI CTEP was conducted under a Cooperative
Research and Development Agreement between NCI and AstraZeneca with
additional support from the Neurofibromatosis Therapeutic
Acceleration Program (NTAP).
Selumetinib
Selumetinib (available in the US under the medicine name
Koselugo) is an inhibitor of mitogen-activated protein kinase
kinases 1 and 2 (MEK1/2).(6) 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 cancers.
Selumetinib 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 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.
AstraZeneca
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 & Metabolism, and Respiratory
& Immunology. Based in Cambridge, UK, 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 .
Contacts
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please click here. For Media contacts, click here.
References
1. 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 February 2020.
2. Hirbe AC, Gutmann DH. Neurofibromatosis type 1: a
multidisciplinary approach to care. The Lancet Neurology.
2014;13:834-43. DOI: 10.1016/S1474-4422(14)70063-8.
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. 2001;68:1110-1118.
5. Evans DGR, O'Hara C, Wilding A, et al. Mortality in
neurofibromatosis 1: in North West England: an assessment of
actuarial survival in a region of the UK since 1989. Eur J Hum
Genet. 2011;19:1187-1191. DOI: 10.1038/ejhg.2011.113.
6. Koselugo (selumetinib) [prescribing information]. Wilmington,
DE: AstraZeneca Pharmaceuticals LP; 2020.
7. Gross A, Wolters P, Dombi E, et al. Selumetinib in Children
with Inoperable Plexiform Neurofibromas. N Engl J Med. 2020;382:
DOI: 10.1056/NEJMoa11912735.
Adrian Kemp
Company Secretary
AstraZeneca PLC
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END
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