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  • Amgen: Nplate – Immune Thrombocytopenia Medication 0 comments
    Oct 11, 2013 5:15 AM | about stocks: AMGN

    Amgen's (NASDAQ:AMGN) Nplate (Romiplostim) is a man-made (recombinant) protein medicine used to treat low blood platelet counts in adults with chronic immune thrombocytopenia (ITP), when certain other medicines, or surgery to remove your spleen, have not worked well enough.

    The Romiplostim molecule was developed by Amgen and is marketed under the trade name Nplate through a restricted usage program called NEXUS. During development and clinical trials the drug was called AMG531.

    (click to enlarge)

    Romiplostim was designated an orphan drug status by the FDA in 2003, since the chronic ITP population in the USA is under 200,000.

    In August, 2008, the FDA approved romiplostim as a long-term treatment for chronic ITP in adults who have not responded to other treatments, such as corticosteroids, intravenous immunoglobulin, Rho(NYSE:D) immune globulin or splenectomy.

    Treatment Regimen

    Romiplostim treatment is generally administered at weekly intervals via subcutaneous injection. Prior to injection, a complete blood count (CBC) is obtained, as the dosage is dependent on the individual's body weight and platelet count at the time of treatment.

    The goal of treatment is to maintain the count above 50,000 per cubic millimeter (mm3) of blood, not to achieve a normal count-defined as 150,000-450,000 per mm3 in most healthy individuals.

    If a count of 200,000 or higher is achieved for two consecutive weeks a reduced dose is administered or treatment is suspended until the count decreases below 200,000.

    Clinical Efficacy

    In well designed, 24-week, Phase III trials, romiplostim was significantly more effective than placebo in achieving the primary endpoint of a protocol-defined durable platelet response in nonsplenectomized or splenectomized adults with chronic immune thrombocytopenic purpura.[6]

    Side Effects

    Romiplostim's effect is to stimulate the patient's megakaryocytes to produce platelets at a more rapid than normal rate, thus overwhelming the immune system's ability to destroy them.

    Since doing so involves changes to the bone marrow chemistry, a number of potentially serious side-effects may develop, including myalgia, joint and extremity discomfort, insomnia, thrombocytosis, which may lead to potentially fatal clots, and bone marrow fibrosis.

    Systematic (IUPAC) name

    L-methionyl[human immunogloblin heavy constant gamma 1-(227 C-terminal residues)-peptide (Fc fragment)] fusion protein with 41 amino acids peptide, (7-7′:10,10′)-bisdisulfide dimer.

    Stocks: AMGN
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