THOUSAND OAKS, Calif.,
Nov. 10, 2017 /PRNewswire/
-- Amgen (NASDAQ:AMGN) today announced that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has adopted a positive opinion to expand the current
indication for Nplate® (romiplostim) to include the
treatment of chronic immune (idiopathic) thrombocytopenic purpura
(ITP) for patients one year of age and older who are refractory to
other treatments (e.g., corticosteroids, immunoglobulins).
The positive CHMP opinion was based on five studies evaluating
the safety and efficacy of Nplate in children with ITP, including
four completed studies (a Phase 3, a Phase 1/2 placebo-controlled
study, and two long-term safety and efficacy studies) and one
ongoing long-term safety and efficacy study.
"Children with ITP are at risk for serious bleeding events due
to low platelet counts, and currently, limited therapeutic options
are available to treat this rare disease," said Sean E. Harper, M.D., executive vice president
of Research and Development at Amgen. "We are pleased by today's
positive CHMP opinion and look forward to working with European
regulatory authorities to deliver on our commitment to bring Nplate
to pediatric patients with ITP who are suffering from
thrombocytopenia."
ITP is a rare, serious autoimmune disease characterized by low
platelet counts in the blood (a condition known as
thrombocytopenia) and impaired platelet
production.1,2 European studies report incidence
rates in children between four and five cases per 100,000 children
each year.3 The treatment goal for children with ITP is
to promote a platelet count that maintains appropriate control of
bleeding, improve symptoms and increase the number of
platelets.1,4
The CHMP positive opinion will now be reviewed by
the European Commission (EC), which has the authority to
approve medicines for the European Union (EU). If
approved, a centralized marketing authorization will be granted
that will be valid in the 28 countries that are members of the
EU. Norway, Iceland and Liechtenstein, as
members of the European Economic Area (EEA), will take
corresponding decisions on the basis of the decision of the EC.
About Nplate® (romiplostim)
Nplate is a thrombopoietin (TPO) receptor agonist indicated for
the treatment of low blood platelet counts in adults with chronic
ITP, who had an insufficient response to other medicines or
surgery. Nplate mimics the body's natural thrombopoietin and is
designed to increase platelet counts in patients with chronic
ITP.5
Nplate is the first U.S. Food and Drug Administration
(FDA)-approved treatment specifically for adult chronic ITP.
In the U.S., Nplate is indicated for the treatment of
thrombocytopenia in patients with chronic ITP who have had an
insufficient response to corticosteroids, immunoglobulins or
splenectomy. Nplate is not indicated for the treatment of
thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause
of thrombocytopenia other than chronic ITP. Nplate should be used
only in patients with ITP whose degree of thrombocytopenia and
clinical condition increase the risk for bleeding. Nplate should
not be used in an attempt to normalize platelet counts.
In the EU, Nplate is indicated for the treatment of adult
chronic ITP patients who are refractory to other treatments (e.g.,
corticosteroids, immunoglobulins).
Nplate was named as a recipient of the U.S. Prix Galien 2009
"Best Biotechnology Product" award and also received the 2009 Scrip
Awards for "Best New Drug." Nplate has also been honored with
numerous awards throughout the EU, including a 2010 Prix Galien in
France in the category of "Drugs
for Rare Diseases," and the 2011 Prix Galien in Germany in the category of "Specialist Care."
In September 2010, Nplate was awarded
the 2010 International Prix Galien Award, an award granted every
two years which recognizes the "Best of the Best" selected from
previous national Prix Galien award
recipients.
Nplate is also approved in Canada, Australia, Russia, Mexico, Switzerland, Lichtenstein, Japan, Argentina, Israel, South
Korea, Hong Kong,
Chile, Serbia, Kazakhstan, Malaysia, Singapore, Colombia, Kuwait, Taiwan, South
Africa, Brazil,
Guatemala, Morocco, Ecuador, Macau, Egypt,
Lebanon, Peru and Venezuela. Nplate has received orphan
designation for chronic ITP in the U.S. (2003), the EU (2005),
Switzerland (2005), Japan (2006), Mexico and South
Korea (2010).
For more information about Nplate, please visit
www.Nplate.com.
Important U.S. Nplate® Safety Information
Risk of Progression of Myelodysplastic Syndromes to Acute
Myelogenous Leukemia
- In Nplate® clinical trials of patients with
myelodysplastic syndromes (MDS) and severe thrombocytopenia,
progression from MDS to acute myelogenous leukemia (AML) has been
observed.
- Nplate® is not indicated for the treatment of
thrombocytopenia due to MDS or any cause of thrombocytopenia other
than chronic ITP.
Thrombotic/Thromboembolic Complications
- Thrombotic/thromboembolic complications may result from
increases in platelet counts with Nplate® use. Portal
vein thrombosis has been reported in patients with chronic liver
disease receiving Nplate®.
- To minimize the risk for thrombotic/thromboembolic
complications, do not use Nplate® in an attempt to
normalize platelet counts. Follow the dose adjustment guidelines to
achieve and maintain a platelet count of ≥ 50 x 109/L.
Loss of Response to Nplate®
- Hyporesponsiveness or failure to maintain a platelet response
with Nplate® should prompt a search for causative
factors, including neutralizing antibodies to Nplate®.
- To detect antibody formation, submit blood samples to Amgen
(1-800-772-6436). Amgen will assay these samples for antibodies to
Nplate® and thrombopoietin (TPO).
- Discontinue Nplate® if the platelet count does not
increase to a level sufficient to avoid clinically important
bleeding after 4 weeks at the highest weekly dose of 10
mcg/kg.
Laboratory Monitoring
- Obtain CBCs, including platelet counts, weekly during the dose
adjustment phase of Nplate® therapy and then monthly
following establishment of a stable Nplate® dose.
- Obtain CBCs, including platelet counts, weekly for at least two
weeks following discontinuation of Nplate®.
Adverse Reactions
- In the placebo-controlled trials, headache was the most
commonly reported adverse drug reaction, occurring in 35% of
patients receiving Nplate® and 32% of patients receiving
placebo. Headaches were usually of mild or moderate severity.
- Most common adverse reactions (≥ 5% higher patient incidence in
Nplate® versus placebo) were Arthralgia (26%, 20%),
Dizziness (17%, 0%), Insomnia (16%, 7%), Myalgia (14%, 2%), Pain in
Extremity (13%, 5%), Abdominal Pain (11%, 0%), Shoulder Pain (8%,
0%), Dyspepsia (7%, 0%), and Paresthesia (6%, 0%).
- Nplate® administration may increase the risk for
development or progression of reticulin fiber formation within the
bone marrow. This formation may improve upon discontinuation of
Nplate®. In a clinical trial, one patient with ITP and
hemolytic anemia developed marrow fibrosis with collagen during
Nplate® therapy.
- Women who become pregnant during
Nplate® treatment are encouraged to enroll in
Amgen's Pregnancy Surveillance Program. Patients or their
physicians should call 1‑800‑77‑AMGEN (1‑800‑772‑6436) to
enroll.
Please see full U.S. Prescribing Information and Medication
Guide at www.Nplate.com.
Important EU Nplate® Safety Information
The
EU Summary of Product Characteristics for Nplate® lists
the following Special Warnings and Precautions: reoccurrence of
thrombocytopenia and bleeding after cessation of treatment,
increased bone marrow reticulin, thrombotic/thromboembolic
complications, progression of existing myelodysplastic syndromes
(MDS), medication errors, loss of response to Nplate, and effects
on red and white blood cells.
The most common adverse reactions observed include
hypersensitivity reactions (including cases of rash, urticaria and
angioedema) and headache. As with all therapeutic proteins, there
is a potential for immunogenicity.
Please refer to the Summary of Product Characteristics for
full European prescribing information.
About Amgen
Amgen is committed to unlocking
the potential of biology for patients suffering from serious
illnesses by discovering, developing, manufacturing and delivering
innovative human therapeutics. This approach begins by using tools
like advanced human genetics to unravel the complexities of disease
and understand the fundamentals of human biology.
Amgen focuses on areas of high unmet medical need and
leverages its expertise to strive for solutions that improve health
outcomes and dramatically improve people's lives. A biotechnology
pioneer since 1980, Amgen has grown to be one of the
world's leading independent biotechnology companies, has reached
millions of patients around the world and is developing a pipeline
of medicines with breakaway potential.
For more information, visit www.amgen.com and follow
us on www.twitter.com/amgen.
Forward-Looking Statements
This news release contains
forward-looking statements that are based on the current
expectations and beliefs of Amgen. All statements, other than
statements of historical fact, are statements that could be deemed
forward-looking statements, including estimates of revenues,
operating margins, capital expenditures, cash, other financial
metrics, expected legal, arbitration, political, regulatory or
clinical results or practices, customer and prescriber patterns or
practices, reimbursement activities and outcomes and other such
estimates and results. Forward-looking statements involve
significant risks and uncertainties, including those discussed
below and more fully described in the Securities and Exchange
Commission reports filed by Amgen, including our most recent annual
report on Form 10-K and any subsequent periodic reports on Form
10-Q and current reports on Form 8-K. Unless otherwise noted,
Amgen is providing this information as of the date of this news
release and does not undertake any obligation to update any
forward-looking statements contained in this document as a result
of new information, future events or otherwise.
No forward-looking statement can be guaranteed and actual
results may differ materially from those we project.
Discovery or identification of new product candidates or
development of new indications for existing products cannot be
guaranteed and movement from concept to product is uncertain;
consequently, there can be no guarantee that any particular product
candidate or development of a new indication for an existing
product will be successful and become a commercial product.
Further, preclinical results do not guarantee safe and effective
performance of product candidates in humans. The complexity of the
human body cannot be perfectly, or sometimes, even adequately
modeled by computer or cell culture systems or animal models. The
length of time that it takes for us to complete clinical trials and
obtain regulatory approval for product marketing has in the past
varied and we expect similar variability in the future. Even when
clinical trials are successful, regulatory authorities may question
the sufficiency for approval of the trial endpoints we have
selected. We develop product candidates internally and through
licensing collaborations, partnerships and joint ventures. Product
candidates that are derived from relationships may be subject to
disputes between the parties or may prove to be not as effective or
as safe as we may have believed at the time of entering into such
relationship. Also, we or others could identify safety, side
effects or manufacturing problems with our products, including our
devices, after they are on the market.
Our results may be affected by our ability to successfully
market both new and existing products domestically and
internationally, clinical and regulatory developments involving
current and future products, sales growth of recently launched
products, competition from other products including biosimilars,
difficulties or delays in manufacturing our products and global
economic conditions. In addition, sales of our products are
affected by pricing pressure, political and public scrutiny and
reimbursement policies imposed by third-party payers, including
governments, private insurance plans and managed care providers and
may be affected by regulatory, clinical and guideline developments
and domestic and international trends toward managed care and
healthcare cost containment. Furthermore, our research, testing,
pricing, marketing and other operations are subject to extensive
regulation by domestic and foreign government regulatory
authorities. Our business may be impacted by government
investigations, litigation and product liability claims. In
addition, our business may be impacted by the adoption of new tax
legislation or exposure to additional tax liabilities. If we fail
to meet the compliance obligations in the corporate integrity
agreement between us and the U.S. government, we could become
subject to significant sanctions. Further, while we routinely
obtain patents for our products and technology, the protection
offered by our patents and patent applications may be challenged,
invalidated or circumvented by our competitors, or we may fail to
prevail in present and future intellectual property litigation. We
perform a substantial amount of our commercial manufacturing
activities at a few key facilities, including in Puerto Rico, and also depend on third parties
for a portion of our manufacturing activities, and limits on supply
may constrain sales of certain of our current products and product
candidate development. In addition, we compete with other companies
with respect to many of our marketed products as well as for the
discovery and development of new products. Further, some raw
materials, medical devices and component parts for our products are
supplied by sole third-party suppliers. Certain of our
distributors, customers and payers have substantial purchasing
leverage in their dealings with us. The discovery of significant
problems with a product similar to one of our products that
implicate an entire class of products could have a material adverse
effect on sales of the affected products and on our business and
results of operations. Our efforts to acquire other companies or
products and to integrate the operations of companies we have
acquired may not be successful. We may not be able to access the
capital and credit markets on terms that are favorable to us, or at
all. We are increasingly dependent on information technology
systems, infrastructure and data security. Our stock price is
volatile and may be affected by a number of events. Our business
performance could affect or limit the ability of our Board of
Directors to declare a dividend or our ability to pay a dividend or
repurchase our common stock.
The scientific information discussed in this news release
relating to new indications for our products is preliminary and
investigative and is not part of the labeling approved by the U.S.
Food and Drug Administration for the products. The products are not
approved for the investigational use(s) discussed in this news
release, and no conclusions can or should be drawn regarding the
safety or effectiveness of the products for these uses.
CONTACT:
Amgen, Thousand Oaks
Kristen Davis, 805-447-3008
(media)
Kristen Neese, 805-313-8267
(media)
Arvind Sood, 805-447-1060
(investors)
References:
- Patient. Immune Thrombocytopenia.
http://patient.info/health/immune-thrombocytopenia-leaflet.
Accessed Aug. 29, 2017.
- Cines DB et al. Immune thrombocytopenic purpura. N Engl J
Med. 2002;346:995-1008.
- Segal JB, Powe NR. Prevalence of immune thrombocytopenia:
analyses of administrative data. J Thromb Haemost.
2006;4(11):2377-83.
- US National Institutes of Health. Immune Thrombocytopenia.
http://www.nhlbi.nih.gov/book/export/html/4917. Accessed
Aug. 29, 2017.
- Nplate® (romiplostim) prescribing information,
Amgen.
View original content with
multimedia:http://www.prnewswire.com/news-releases/amgen-receives-positive-chmp-opinion-to-expand-use-of-nplate-romiplostim-in-pediatric-patients-with-chronic-immune-thrombocytopenic-purpura-300553816.html
SOURCE Amgen