Nplate(R) Approved in the European Union for the Treatment of Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
First and Only Approved Platelet Producer in Europe Represents New
Treatment Approach for Serious Chronic Autoimmune Disorder
ZUG, Switzerland, Feb. 6 /PRNewswire-FirstCall/ -- Amgen (Nasdaq: AMGN)
today announced that the European Commission (EC) has granted marketing
authorisation for Nplate(R) (romiplostim) for the treatment of splenectomised
adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) patients who
are refractory to other treatments (e.g. corticosteroids, immunoglobulins).
Nplate may be considered as second line treatment for adult non-splenectomised
ITP patients where surgery is contra-indicated.
Nplate, the first and only approved platelet producer in Europe, has been
granted marketing authorisation for the European Union (EU) based upon a
positive opinion from the European Committee for Medicinal Products for Human
Use in November 2008. Nplate works by raising and sustaining platelet counts,
representing a novel approach for the long-term treatment of this chronic
Chronic ITP is a serious autoimmune disorder characterised by low platelet
counts in the blood (thrombocytopenia), which can lead to serious bleeding
events. ITP is recognised as an orphan disease by the European Medicines
Agency (EMEA) and there are an estimated 50,000 adult patients with chronic
ITP in the EU.(1) Nplate is a breakthrough thrombopoiesis-stimulating
peptibody that represents a new approach to the management of ITP by
increasing platelet production and potentially reducing the need for rescue
and other maintenance medications.
"Nplate is the first approved long-term treatment option in Europe that
specifically targets platelet production," said Dr Roberto Stasi, Department
of Medical Sciences, Regina Apostolorum Hospital, Italy. "Although ITP affects
a limited patient population, it can have a significant impact on patient
lives. Nplate represents a potentially high value option for these patients."
The EU approval of Nplate is based on data from two separate
placebo-controlled Phase 3 studies, demonstrating that platelet counts were
raised and sustained in 83 percent of patients for both splenectomised and
non-splenectomised groups when treated with Nplate. Additionally, patients
treated with Nplate were able to reduce or discontinue concomitant medications
such as corticosteroids which are often not well tolerated. Nplate patients
also used far less "emergency" medications such as IVIG and Win-Rho, whose
effects are transient.
Upon completion of the Phase 3 studies almost 90 percent of patients
elected to subsequently enroll into the Nplate long term extension study which
demonstrated that Nplate continued to effectively increase and sustain
platelet counts. In this open label long term extension study the average
treatment period was 76 weeks and the longest duration of treatment was 204
Key findings from the extension study showed platelet counts of
Nplate-treated patients were increased from baseline by 20,000 platelets per
microliter more than 80 percent of the time in 47 percent of patients and more
than half the time in 67 percent of patients.
"Amgen is committed to advancing the discovery and development of new
therapies for grievous illnesses where there is an unmet need," said Willard
Dere, M.D., senior vice president and international chief medical officer at
Amgen. "The European approval of Nplate is the result of more than 15 years of
research and represents an important biotechnology milestone as it is the
first approved peptibody, an innovative platform for delivering targeted
About Adult ITP
In patients with ITP, platelets - or blood elements needed to prevent
bleeding - are destroyed by the patient's own immune system. Low platelet
counts leave adult ITP patients open to sudden serious bleeding events. The
risk for serious bleeding events increases when platelet counts drop to less
than 30,000 platelets per microliter; normal counts range from 150,000 to
400,000 platelets per microliter. ITP has historically been considered a
disease of platelet destruction although recent data suggest that the body's
natural platelet production processes in ITP are unable to compensate for low
levels of platelets in the blood. Increasing the rate of platelet production
may address low platelet levels associated with ITP.
Currently available treatments (i.e., corticosteroids, immunoglobulins)
have limited application due to poor tolerability or transient effects.
Surgical therapy (removal of the spleen) is also available to adult patients
with chronic ITP, but does not work in all cases. Currently, there are
140,000 treated chronic ITP patients in Europe and the U.S. ITP affects about
twice as many adult women as men.
Nplate was granted approval for ITP by the regulatory bodies in Australia
in July and the United States (U.S.) in August 2008. Amgen has filed for
regulatory approval of Nplate in Canada and Switzerland and these applications
are currently under review. Nplate has also received orphan designation for
ITP in the U.S. (2003), the EU (2005), Switzerland (2005) and Japan (2006).
Nplate is the first treatment specifically developed for ITP. It is also
being investigated for potential use in paediatric ITP, myelodysplastic
syndromes (MDS) and chemotherapy-induced thrombocytopenia (CIT).
Important EU Nplate Safety Information
The most common side effects are headache, fatigue, arthralgia, myalgia,
injection site bruising, injection site pain, peripheral oedema, dizziness,
muscle spasms, nausea, contusion, diarrhoea, bone marrow disorder, influenza
like illness, insomnia and pruritus.
Reoccurrence of thrombocytopenia and bleeding after cessation of treatment
and increased bone marrow reticulin have been associated with romiplostim
treatment in the clinical trials. Thrombotic/thromboembolic complications,
progression of existing haematopoietic malignancies or myelodysplastic
syndromes (MDS), and effects on red and white blood cells are all potential
risks associated with romiplostim treatment. As with all therapeutic proteins,
patients may develop antibodies to the therapeutic protein.
Important U.S. Nplate Safety Information
Serious adverse reactions associated with Nplate in clinical studies were
bone marrow reticulin deposition and worsening thrombocytopenia after Nplate
discontinuation. Additional risks include bone marrow fibrosis,
thrombotic/thromboembolic complications, lack or loss of response to Nplate,
and haematological malignancies and progression of malignancy in patients with
a pre-existing haematological malignancy or myelodysplastic syndromes (MDS).
Nplate is not indicated for the treatment of thrombocytopenia due to MDS or
any cause of thrombocytopenia other than chronic ITP.
In the U.S. Nplate is available only through a restricted distribution
program called Nplate(TM) NEXUS (Network of Experts Understanding and
Supporting Nplate and Patients) Program.
In the placebo-controlled studies, headache was the most commonly reported
adverse drug reaction.
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(1) Gernsheimer. T, Epidemiology and pathophysiology of immune
thrombocytopenic purpura. European Journal of Haematology. 2008; 80:3-8
CONTACT: Amgen, Thousand Oaks
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