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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 disease.
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 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.
"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 therapies."
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.
About Nplate
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.
About Amgen
Amgen discovers, develops, manufactures and delivers innovative human therapeutics. A biotechnology pioneer since 1980, Amgen was one of the first companies to realize the new science's promise by bringing safe and effective medicines from lab, to manufacturing plant, to patient. Amgen therapeutics have changed the practice of medicine, helping millions of people around the world in the fight against cancer, kidney disorder, rheumatoid arthritis, and other serious illnesses. With a deep and broad pipeline of potential new medicines, Amgen remains committed to advancing science to dramatically improve people's lives. To learn more about our pioneering science and our vital medicines, visit www.amgen.com.
Forward-Looking Statement
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