THOUSAND OAKS, Calif.,
April 19, 2016 /PRNewswire/ --
Amgen (NASDAQ:AMGN) today announced that The Lancet
published results from a Phase 3 randomized, double-blind,
placebo-controlled study of Nplate® (romiplostim) in
children with symptomatic immune thrombocytopenia (ITP). The study
showed that 52 percent of Nplate patients achieved a durable
platelet response, compared with 10 percent of placebo-treated
patients (p=0.002, odds ratio 9.1, 95 percent CI: 1.9,
43.2).
"Children with ITP are at risk for serious bleeding events due
to low platelet counts, which can be very frightening for these
children and their parents," said Michael
D. Tarantino, M.D., The Bleeding and Clotting Disorders
Institute, professor of Pediatrics and Medicine, University of Illinois College of Medicine-Peoria,
Peoria, Illinois. "The results of
this study suggest that romiplostim could reduce the frequency and
severity of bleeding events for children suffering from symptomatic
ITP, thus providing them with another potential treatment
option."
The study met the primary endpoint of durable platelet response
and showed that children who were treated with Nplate had increased
rates of overall platelet response, and patients who responded to
Nplate maintained consistently elevated platelet counts. These
findings demonstrate that Nplate may be a potential treatment
option for children with symptomatic ITP of more than six months
duration. The most frequently reported adverse events (AEs)
included contusion, epistaxis, headache and upper respiratory tract
infections. The overall safety profile observed in the Nplate arm
was similar to the known safety profile of Nplate.
"Nplate helps bone marrow produce more platelets, which in turn
helps prevent bruising and bleeding which is important for children
faced with this condition. These data are important in
understanding how Nplate may play a role in helping children manage
this disease," said Sean E. Harper,
M.D., executive vice president of Research and Development at
Amgen. "We will work with regulatory
authorities towards an approval for Nplate for pediatric
patients."
The treatment goal for children with ITP is to promote a
platelet count that maintains appropriate control of
bleeding,1 improve symptoms and increase the number of
platelets.2
ITP is a rare, serious autoimmune disease characterized by low
platelet counts in the blood (a condition known as
thrombocytopenia) and impaired platelet production.2,3
In the United States (U.S.), an
average estimate of the incidence in children is 5 cases in 1,000
each year.4
About the Phase 3 Study
This Phase 3 double-blind study randomized 62 children who have had
ITP for more than six months to weekly Nplate or placebo (2:1) for
24 weeks. Durable platelet response, the primary endpoint of the
study, was defined as achieving weekly platelet responses
(increased platelets) without rescue medication in at least six of
the final eight weeks.
Secondary endpoints of the study included the evaluation of
overall platelet response, the total number of weekly platelet
responses, the use of ITP rescue medications, composite bleeding
episodes and the overall safety of Nplate. Exploratory endpoints
included the evaluation of bleeding incidence and changes in
patient reported outcomes. Rescue medication was defined as any
medication intended to increase platelet counts or prevent
bleeding, and any increase in dose, frequency or additional therapy
was categorized as rescue medication. Patients entering the study
were permitted to use the same standard-of-care therapy, dose and
schedule from when screening platelet counts were
measured.
By the final eight weeks of the study, noncutaneous bleeding had
decreased with Nplate, and rates of durable platelet response were
52 percent compared to 10 percent with placebo (p=0.002,
odds ratio 9.1, 95 percent CI: 1.9, 43.2). Rates of overall
platelet response with Nplate were 71 percent (30/42) compared with
20 percent with placebo (p=0.0002, odds ratio 9.0, 95
percent CI: 2.5, 32.3), and rates of any platelet response were 81
percent (34/42) with Nplate compared to 55 percent (11/20) with
placebo (p=0.0313).
The overall safety profile on the pediatric subjects who
received Nplate in this study was similar to the known safety
profile of Nplate. The most frequently reported AEs included
contusion, epistaxis, headache and upper respiratory tract
infections. Oropharyngeal pain occurred more frequently with Nplate
[26.2 percent (11/42) vs. 5.3 percent (1/19) in placebo-treated
patients]; of the 11 patients treated with Nplate with
oropharyngeal pain, streptococcal pharyngitis (n=2), allergic
rhinitis (n=2), gastroesophageal reflux (n=1) and serum sickness
from IVIg (n=1) were also reported. No oropharyngeal pain AEs were
serious or considered treatment-related. No patients died and none
withdrew due to AEs.
Serious adverse events (SAEs) were seen in 23.8 percent of
Nplate patients and 5.3 percent of placebo patients. SAEs seen in
the Nplate arm included epistaxis, contusion and headache (n=2
each), bronchiolitis, nausea, petechiae, epilepsy, fever,
thrombocytosis, urinary tract infection and vomiting (n=1 each).
One subject with treatment-related SAEs experienced headache and
thrombocytosis, which did not recur when romiplostim was restarted.
There were no thrombotic events reported in the study.
About Nplate® (romiplostim)
Nplate is a thrombopoietin receptor agonist indicated for the
treatment of low blood platelet counts in adults with chronic
immune thrombocytopenia (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 FDA-approved treatment specifically for
adult chronic ITP. It is also being investigated for potential use
in children ages 12 months to 18 years old with
chemotherapy-induced thrombocytopenia.
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 European Union (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. Safety Information Regarding Nplate
(romiplostim)
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.
Please see full Prescribing Information for Nplate 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 MDS (in patients with 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, urticarial and
angioedema) and headache. As with all therapeutic proteins, there
is a potential for immunogenicity.
About Amgen's Commitment to Oncology
Amgen Oncology is committed to helping patients take on some of the
toughest cancers, such as those that have been resistant to drugs,
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and our targeted medicines and immunotherapies focus on more than a
dozen different malignancies, ranging from blood cancers to solid
tumors. With decades of experience providing therapies for cancer
patients, Amgen continues to grow its portfolio of innovative and
biosimilar oncology medicines.
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
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Amgen focuses on areas of high unmet medical need and leverages
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For more information, visit www.amgen.com and follow us on
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CONTACT: Amgen, Thousand
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References:
- US National Institutes of Health. Immune Thrombocytopenia.
http://www.nhlbi.nih.gov/book/export/html/4917. Accessed
Feb. 4, 2016.
- http://patient.info/health/immune-thrombocytopenia-leaflet.
Accessed Mar 16, 2016
- Cines DB et al. Immune thrombocytopenic purpura. N Engl J
Med. 2002; 346:995-1008.
- Platelet Disorder Support Association. About ITP.
http://www.pdsa.org/about-itp.html. Accessed Feb. 4, 2016.
- Fogarty PF et al. The epidemiology of immune thrombocytopenic
purpura. Curr Opin Hematol. 2007;14(5):515-519.
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