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  • Oxlumo (lumasiran) 94.5mg/0.5ml

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    Description

    Oxlumo (Lumasiran) is an innovative RNA interference (RNAi) therapeutic specifically designed for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic condition. PH1 causes the overproduction of oxalate, which can lead to the formation of kidney stones and eventual kidney failure. By reducing the levels of oxalate, Oxlumo helps prevent kidney damage and other complications associated with PH1.

    How Oxlumo (Lumasiran) Works

    Oxlumo works by targeting the HAO1 gene that encodes glycolate oxidase (GO), an enzyme responsible for the production of oxalate in the liver. Lumasiran is a small interfering RNA (siRNA) that selectively silences the HAO1 gene, effectively reducing the production of glycolate oxidase. As a result, the production of oxalate is significantly decreased, which in turn lowers the risk of kidney stone formation and other complications in patients with primary hyperoxaluria type 1. Oxlumo is administered via subcutaneous injection and works by leveraging the body’s natural RNAi mechanisms to inhibit the disease process at the genetic level.

    Indications

    Oxlumo is indicated for the following:
    • Treatment of Primary Hyperoxaluria Type 1 (PH1): Oxlumo is approved for the treatment of PH1, a rare genetic disorder that causes excess oxalate production, leading to kidney stones, nephrocalcinosis, and kidney failure.

    Side Effects

    Common side effects of Oxlumo include:
    • Injection site reactions (redness, swelling, pain)
    • Abdominal pain
    • Diarrhea
    • Headache
    • Nausea
    • Fatigue
    • Fever
    Serious side effects may include:
    • Elevated liver enzymes
    • Hypersensitivity reactions
    • Severe injection site reactions (rare)

    Contraindications

    Oxlumo is contraindicated in the following conditions:
    • Patients with known hypersensitivity to Lumasiran or any of the excipients in Oxlumo
    • Pregnancy (as safety has not been well established in pregnant women)
    • Severe liver disease (as it may exacerbate liver-related complications)
    • Patients who are breastfeeding, as it is unknown if Lumasiran passes into breast milk

    Price of Oxlumo (Lumasiran) in Different Countries

    The price of Oxlumo varies based on country-specific healthcare systems, insurance coverage, and region. Below is a table showing the approximate cost of Oxlumo in different countries:
    Country Price (per year) Source
    United States $450,000 per year GoodRx
    United Kingdom £350,000 per year NICE BNF
    Germany €420,000 per year Medikamentenpreis
    Canada CAD 600,000 per year DrugBank
    Australia AUD 650,000 per year PBS Australia

    Top 5 Global Brands for Genetic Disorder Treatments

    While Oxlumo is a breakthrough treatment for primary hyperoxaluria, several other global brands are leading the way in treating rare genetic disorders. These include:
    • Oxlumo (Lumasiran) by Alnylam Pharmaceuticals
    • Spinraza (Nusinersen) by Biogen for spinal muscular atrophy (SMA)
    • Kalydeco (Ivacaftor) by Vertex Pharmaceuticals for cystic fibrosis
    • Luxturna (Voretigene neparvovec) by Spark Therapeutics for retinal dystrophy
    • Brineura (Cerliponase alfa) by BioMarin for Batten disease
     
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    Oxlumo (lumasiran) 94.5mg/0.5ml
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