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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
Nplate, the first and only approved platelet producer in
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
"Nplate is the first approved long-term treatment option in
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 weeks.
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.
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
Nplate was granted approval for ITP by the regulatory bodies in
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
Sabeena Ahmad, +41 41-3692-530 (EU media, oncology)
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