YASTEST
New Formulation of
ONCASPAR®
(pegaspargase) Receives Positive CHMP Opinion in Europe for
Patients with Acute Lymphoblastic Leukemia (ALL)
If approved, lyophilized formulation would offer three
times longer shelf life vs. liquid ONCASPAR, enabling better supply
management and improved product availability
Zug, Switzerland - October
13, 2017 - Shire plc (LSE: SHP,
NASDAQ: SHPG), the global leader in rare diseases, today announced
that the Committee for Medicinal Products for Human Use (CHMP) of
the European Medicines Agency (EMA) has adopted a positive opinion
recommending the marketing authorization for lyophilized ONCASPAR
(pegaspargase), as a component of antineoplastic combination
therapy in acute lymphoblastic leukemia (ALL) in pediatric patients
from birth to 18 years, and in adult patients. Lyophilized ONCASPAR
is a freeze-dried formulation of ONCASPAR. The liquid form of
ONCASPAR is currently approved for the same indication in ALL, and
is part of the pediatric standard therapy in ALL in many European
countries.[1] The CHMP's
positive opinion will be submitted to the European Commission (EC),
which is responsible for granting marketing authorizations for
medicines in the EU.
Acute lymphoblastic leukemia (ALL) is a cancer of
the white blood cells and is characterized by an overproduction and
accumulation of lymphoblasts, immature white blood cells. ALL is
the most common type (~75%) of cancer among children diagnosed with
leukemia.[2] ALL can be
curable within certain pediatric patient populations, with a 5-year
survival rate of more than 90% in children treated with regimens
including ONCASPAR.[3],[4],[5]
"Lyophilized ONCASPAR builds on more than a decade
of data and research with liquid ONCASPAR, and with no change in
dosing regimen, it offers a three-times longer shelf life," said
Howard B. Mayer, M.D., SVP and ad-interim Head, Global Research and
Development, Shire. "Prolonging shelf life to 24 months for this
critically-important therapy facilitates management of product
inventory by enabling greater flexibility and longer-term planning.
Once approved, with the extended shelf life of lyophilized
ONCASPAR, we also hope to improve access to the medicine for ALL
patients in countries currently not offering liquid ONCASPAR."
Lyophilized ONCASPAR works the same way as the
liquid formulation by rapidly depleting serum L-asparagine levels
and interfering with protein synthesis, thereby depriving
lymphoblasts of asparaginase and resulting in cell death. That is
why asparaginase is a critical component of the treatment regimen
for ALL patients as it is a proven approach to inducing leukemic
cell death.4,[6],[7],[8],[9],[10],[11]
About Lyophilized
ONCASPAR
Lyophilized ONCASPAR builds on more than a decade of data and
research with liquid ONCASPAR, a pegylated asparaginase. The
positive opinion is based on analytical and nonclinical studies,
which demonstrate that the lyophilized formulation of ONCASPAR is
comparable to the liquid formulation. Once reconstituted,
lyophilized ONCASPAR demonstrates similar pharmacokinetic
(PK)/pharmacodynamics (PD) to liquid ONCASPAR.1 The new
lyophilized formulation offers no change in dosing regimen but a
longer shelf life that is three times that of the liquid
formation.1
About ONCASPAR
In Europe, ONCASPAR is indicated as a component of antineoplastic
combination therapy in acute lymphoblastic leukemia (ALL) in
pediatric patients from birth to 18 years, and adult patients.
ONCASPAR can be given by intramuscular injection
or intravenous infusion. For smaller volumes of ONCASPAR, the
preferred route of administration is intramuscular. When ONCASPAR
is given by intramuscular injection the volume injected at one site
should not exceed 2 ml in children and adolescents and 3 ml in
adults. If higher volume is given, the dose should be divided and
given at several injection sites. Intravenous infusion of ONCASPAR
is usually given over a period of 1 to 2 hours in
100 ml sodium chloride 9 mg/ml (0.9%) solution for
injection or 5% glucose solution. The diluted solution of ONCASPAR
can be given together with an already-running infusion of either
sodium chloride 9 mg/ml or 5% glucose. Do not infuse other
medicinal products through the same intravenous line during
administration of ONCASPAR.
Safety Information
ONCASPAR is contraindicated in patients with hypersensitivity to
the active substance or to any of the excipients, in patients with
severe hepatic impairment, and in patients with a history of
serious thrombosis, pancreatitis, or serious haemorrhagic events
with prior L-asparaginase therapy.
Anaphylaxis or serious allergic reactions can
occur; therefore, patients should be observed for 1 hour after
administration having resuscitation equipment ready. Discontinue
ONCASPAR in patients with serious allergic reactions. There have
been reports of adverse reactions of pancreatitis. If pancreatitis
is suspected ONCASPAR should be discontinued. ONCASPAR should also
be discontinued in patients with serious thrombotic events.
Combination therapy with ONCASPAR can result in
hepatic toxicity and central nervous system toxicity. Appropriate
monitoring should be performed for liver impairment, blood and
urine glucose levels as well as serum amylase for early signs of
inflammation of the pancreas.
Very common adverse reactions reported in clinical
trial data and the post-marketing experience of ONCASPAR in ALL
patients include: hyperglycemia, pancreatitis, diarrhea, abdominal
pain, nausea, vomiting, stomatitis, hypersensitivity, urticaria,
anaphylactic reaction, weight decreased, decreased appetite, and
rash.
Common adverse reactions include: febrile
neutropenia, anemia, coagulopathy, hepatotoxicity, fatty liver,
infections, sepsis, amylase increased, alanine aminotransferase
increased, blood bilirubin increased, blood albumin decreased,
neutrophil count decreased, platelet count decreased, activated
partial thromboplastin time prolonged, prothrombin time prolonged,
hypofibrinogenemia, hypertriglyceridemia, hyperlipidemia,
hypercholesterolemia, pain in extremities, seizure, peripheral
motor neuropathy, syncope, posterior reversible leukoencephalopathy
syndrome, hypoxia, and thrombosis.1
For Further Information, Please
Contact
Investor Relations |
|
|
Ian Karp |
ikarp@shire.com |
+1 781 482 9018 |
Robert Coates |
rcoates@shire.com |
+44 1256 894874 |
|
|
|
Media |
|
|
Annabel Cowper |
annabel.cowper@shire.com |
+41 44 878 6638 |
Gwen Fisher |
gfisher@shire.com |
+1 781 482 9649 |
NOTES TO EDITOR
About Shire
Shire is the leading global biotechnology company focused on
serving people with rare diseases. We strive to develop
best-in-class products, many of which are available in more than
100 countries, across core therapeutic areas including Hematology,
Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders,
Gastrointestinal / Internal Medicine / Endocrine and Hereditary
Angioedema; and a growing franchise in Oncology.
Our employees come to work every day with a shared
mission: to develop and deliver breakthrough therapies for the
hundreds of millions of people in the world affected by rare
diseases and other high-need conditions, and who lack effective
therapies to live their lives to the fullest.
www.shire.com
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Reports on Form 10-Q, in each case including those risks outlined
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[1] Lyophilized
Oncaspar SMPC reference
[2] American
Cancer Society.
https://www.cancer.org/cancer/leukemia-in-children/about/what-is-childhood-leukemia.html
[3] American
Cancer Society.
http://www.cancer.org/cancer/leukemiainchildren/detailedguide/childhood-leukemia-survival-rates
[4] Angiolillo
AL, Schore RJ, Devidas M, et. al. Pharmacokinetic and
pharmacodynamic properties of calaspargase pegol Escherichia coli
L-asparaginase in the treatment of patients with acute
lymphoblastic leukemia: results from Children's Oncology Group
Study AALL07P4. J Clin Oncol. 2014 Dec 1;32(34):3874-82. doi:
10.1200/JCO.2014.55.5763. Epub 2014 Oct 27.
[5] Place AE,
et al. Lancet Oncol 2015; 16: 1677-1690 +
appendix.
[6] Oncaspar
(pegaspargase) Summary of Product Characteristics. (SmPC) EU
1/2016.
[7] Wilson GJ,
et al. Am J Physiol Endocrinol Metab 2013; 305: E1124-1133.
[8] Story MD,
et al. Cancer Chemother Pharmacol 1993; 32: 129-133.
[9] Avramis VA
and Panosyan EH, Clin Pharmacokinet 2005; 44: 367-393.
[10] Schwarz
JH, et al. Biochemistry 1966; 56: 1516-1519.
[11] Graham ML,
Adv Drug Deliv Rev 2003; 55: 1293-302.
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The issuer of this announcement warrants that they are solely
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information contained therein.
Source: Shire plc via Globenewswire
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