Nplate(TM) (Romiplostim) Receives Positive Opinion for Marketing Authorisation in the European Union
First and Only Approved Platelet Producer Represents New Treatment Approach
for Serious Chronic Autoimmune Disorder
THOUSAND OAKS, Calif., Nov. 21 /PRNewswire-FirstCall/ -- Amgen
(Nasdaq: AMGN) today announced that the European Committee for Medicinal
Products for Human Use (CHMP) has issued a positive opinion recommending
marketing authorisation for Nplate(TM) (romiplostim) in the European Union
(EU). The CHMP recommends Nplate for adult chronic immune (idiopathic)
thrombocytopenia purpura (ITP) splenectomised patients who are refractory to
other treatments (e.g. corticosteroids, immunoglobulins). Nplate may be
considered as second line treatment for adult non-splenectomised patients
where surgery is contra-indicated.
"Nplate will address an unmet medical need for thousands of patients in
the European Union as it is a unique treatment option that increases platelet
production and avoids immune suppression in adult chronic ITP patients," said
Willard Dere, M.D., senior vice president and international chief medical
officer at Amgen.
The novel peptibody technology upon which romiplostim is based represents
a promising new approach for treating adult patients with chronic ITP, an
autoimmune disorder affecting an estimated 50,000 people in the EU, which can
lead to serious bleeding events that can be potentially life threatening.
Nplate, a thrombopoietin (TPO) mimetic, is a novel engineered therapeutic
fusion protein with attributes of both peptides and antibodies, but is
distinct from each. Nplate works similarly to TPO, a natural protein in the
body. Nplate stimulates the TPO receptor, which is necessary for growth and
maturation of bone marrow cells that produce platelets.
The CHMP positive opinion 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 ITP and
emergency medications which are often not well tolerated or whose effects are
transient (i.e. corticosteroids, IVIG, Win-Rho Anti-D therapy).
Upon completion of the Phase 3 studies almost 90 percent of patients
elected to subsequently enroll into the romiplostim long term extension study
which demonstrated that, after three years, Nplate continued to effectively
increase and sustain platelet counts. In this open label long term extension
study some patients were treated for over 156 weeks and in the interim
analysis the median treatment duration in this study is 65 weeks.
About Adult ITP
In patients with ITP, platelets -- or blood cells 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, making
it impossible to arrest blood flow. 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. In addition to the
European Union (EU), 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 pediatric ITP, myelodysplastic
syndrome (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, oedema peripheral, dizziness,
muscle spasms, nausea, contusion, diarrhea, 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 hematological malignancies and progression of malignancy in patients with
a pre-existing hematological malignancy or Myelodysplastic Syndrome (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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