THOUSAND OAKS, Calif.,
Oct. 18, 2019 /PRNewswire/ -- Amgen
(NASDAQ:AMGN) today announced that the U.S. Food and Drug
Administration (FDA) approved Amgen's Supplemental Biologics
License Application (sBLA) for Nplate® (romiplostim) to
include new data in its U.S. prescribing information showing
sustained platelet responses in adults with immune thrombocytopenia
(ITP), a rare, serious autoimmune disease characterized by low
platelet counts. The updated indication expands treatment with
Nplate to newly diagnosed and persistent adult ITP patients who
have had an insufficient response to corticosteroids,
immunoglobulins or splenectomy. In December of last year, the FDA
approved another sBLA for Nplate in the treatment of pediatric
patients with ITP.
"These new data are the first of their kind to prospectively
examine treatment-free remission as an outcome for patients with
ITP. Thirty-two percent of patients who received Nplate soon after
an insufficient response to the first course of steroids maintained
platelet response for at least six months without Nplate or any
other ITP therapy," said David M.
Reese, M.D., executive vice president of Research and
Development at Amgen. "This approval will provide patients the
opportunity to receive Nplate earlier in the course of their
disease, potentially reducing their need for prolonged steroid use.
We are excited to make Nplate available to more patients with this
rare blood disorder."
The sBLA was based on an open-label, single-arm Phase 2 trial of
adults with ITP diagnosed ≤ 6 months prior who had an insufficient
response to first-line treatment, including corticosteroids (N=75).
The median time from ITP diagnosis to study enrollment was 2.2
months. On the primary endpoint, the median number of months with
platelet response (≥ 50 x 109/L) was 11 months during
the 12-month treatment period (95% CI: 10, 11), with a median time
to first platelet response of 2.1 weeks (95% CI: 1.1, 3.0).
Additionally, 93% (70) of patients achieved one or more platelet
responses during the 12-month treatment period. On the secondary
endpoint, 32% (24) of patients achieved remission for at least six
months, defined by maintaining a platelet count ≥ 50 x
109/L in the absence of Nplate and any medication for
ITP (concomitant or rescue).
"Among adults with immune thrombocytopenia, there is a need for
treatment options that can get patients to sustained remission,"
said Caroline Kruse, president and
chief executive officer, Platelet Disorder Support Association.
"The addition of this new data will help physicians and patients
communicate and weigh the benefits and risks of treatment to find
an appropriate treatment choice."
The safety profile of Nplate was similar across patients,
regardless of ITP duration. The following adverse reactions (at
least 5% incidence and at least 5% more frequent with Nplate
compared with placebo or standard of care) occurred in Nplate
patients with ITP duration up to 12 months: bronchitis, sinusitis,
vomiting, arthralgia, myalgia, headache, dizziness, diarrhea, upper
respiratory tract infection, cough, nausea and oropharyngeal pain.
The adverse reaction of thrombocytosis occurred with an incidence
of 2% in adults with ITP duration up to 12 months.
About the Phase 2 Study
The Phase 2 study was a
single-arm, open-label study designed to assess the safety and
efficacy of Nplate in adult patients who had an insufficient
response (platelet count ≤ 30 x 109/L) to first line
therapy (N=75). The median time from ITP diagnosis to study
enrollment was 2.2 months. Prior ITP treatments included
corticosteroids, immunoglobulins and anti-D immunoglobulins. Rescue
therapies were permitted. Patients received single weekly SC
injections of Nplate over a 12-month treatment period, with
individual dose adjustments to maintain platelet counts (50 x
109/L to 200 x 109/L).
About Immune Thrombocytopenia (ITP)
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 In the U.S., the
estimated incidence of ITP is 6.1 per 100,000 adults
annually.2 Nearly 20,000 people are newly diagnosed with
ITP each year in the U.S.2
About Nplate® (romiplostim)
Nplate is
a thrombopoietin (TPO) receptor agonist that mimics the body's
natural TPO and is designed to increase platelet counts in patients
with ITP.3
In the U.S:
- Nplate is approved for the treatment of thrombocytopenia in
adult patients with ITP who have had an insufficient response to
corticosteroids, immunoglobulins, or splenectomy.
- Nplate is approved for the treatment of thrombocytopenia in
pediatric patients 1 year of age and older with ITP for at least 6
months who have had an insufficient response to corticosteroids,
immunoglobulins, or splenectomy.
In the European Union (EU):
- Nplate is approved for the treatment of chronic ITP in adults
and in children age one year and older with ITP for at least six
months, who have had an insufficient response to other medicines or
had surgery to remove the spleen.
Nplate is also approved in 69 countries,
including Canada and Australia.
For more information about Nplate, please
visit www.Nplate.com.
IMPORTANT SAFETY INFORMATION
Risk of Progression of Myelodysplastic Syndromes to Acute
Myelogenous Leukemia
- In Nplate® (romiplostim) 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 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.
Adverse Reactions
Adult ITP
- In the placebo-controlled trials of adult ITP patients,
headache was the most commonly reported adverse drug reaction,
occurring in 35% of patients receiving Nplate® and 32%
of patients receiving placebo. Adverse drug reactions in adults
with a ≥ 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%).
- The safety profile of Nplate was similar across patients,
regardless of ITP duration. The following adverse reactions (at
least 5% incidence and at least 5% more frequent with Nplate
compared with placebo or standard of care) occurred in Nplate
patients with ITP duration up to 12 months: bronchitis, sinusitis,
vomiting, arthralgia, myalgia, headache, dizziness, diarrhea, upper
respiratory tract infection, cough, nausea and oropharyngeal pain.
The adverse reaction of thrombocytosis occurred with an incidence
of 2% in adults with ITP duration up to 12 months.
Pediatric ITP
- The most common adverse reactions experienced by ≥ 5% of
patients receiving Nplate with > 5% higher incidence in the
romiplostim arm across the two placebo-controlled trials were
contusion (41%), upper respiratory tract infection (31%),
oropharyngeal pain (25%), pyrexia (24%), diarrhea (20%), rash
(15%), and upper abdominal pain (14%).
- In pediatric patients of age > 1 year receiving romiplostim
for ITP, adverse reactions with an incidence of > 25% in the two
randomized trials were: contusion (41%), upper respiratory tract
infection (31%), and oropharyngeal pain (25%).
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.
INDICATIONS
Nplate® is a thrombopoietin
receptor agonist indicated for the treatment of thrombocytopenia in
adult patients with immune thrombocytopenia (ITP) who have had an
insufficient response to corticosteroids, immunoglobulins, or
splenectomy. Nplate® is indicated for the treatment
of thrombocytopenia in pediatric patients 1 year of age and older
with ITP for at least 6 months 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 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.
Please see full Prescribing Information and Medication
Guide.
About Amgen Oncology
Amgen is searching for and
finding answers to incredibly complex questions that will advance
care and improve lives for cancer patients and their families. Our
research drives us to understand the disease in the context of the
patient's life – not just their cancer journey – so they can take
control of their lives.
For the last four decades, we have been dedicated to discovering
the firsts that matter in oncology and to finding ways to reduce
the burden of cancer. Building on our heritage, Amgen continues to
advance the largest pipeline in the Company's history, moving with
great speed to advance those innovations for the patients who need
them.
At Amgen, we are driven by our commitment to transform the lives
of cancer patients and keep them at the center of everything we
do.
For more information, follow us on
www.twitter.com/amgenoncology.
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
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CONTACT: Amgen, Thousand
Oaks
Trish Hawkins, 805-447-5631
(Media)
Jessica Akopyan, 805-447-0974
(Media)
Arvind Sood, 805-447-1060
(Investors)
References
- National Organization for Rare Disorders. Immune
Thrombocytopenia. https://rarediseases.org/rare-diseases/immune-thrombocytopenia/.
Accessed on: October 10, 2019.
- Weycker D, Hanau A, Hatfield M, et al. Primary immune
thrombocytopenia in US clinical practice: incidence and healthcare
burden in first 12 months following diagnosis. J Med Econ.
2019 Oct 9:1-9.
- Nplate® (romiplostim) prescribing
information, Amgen.
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