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  • Waylivra (volanesorsen) injection

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    Description

    Waylivra, containing the active ingredient Volanesorsen, is an injectable medication used to treat patients with familial chylomicronemia syndrome (FCS). FCS is a rare genetic disorder that impairs the body’s ability to break down fats, leading to extremely high levels of triglycerides in the blood, which increases the risk of recurrent and severe pancreatitis. Waylivra is specifically designed to lower triglyceride levels in these patients by targeting the production of apolipoprotein C-III (ApoC-III), a key regulator of triglyceride metabolism.

    How Waylivra (Volanesorsen) Works

    Waylivra works through an antisense oligonucleotide mechanism. Volanesorsen is a short strand of chemically modified RNA that binds to the messenger RNA (mRNA) responsible for the production of apolipoprotein C-III (ApoC-III). By binding to this mRNA, Waylivra inhibits the translation and production of ApoC-III. ApoC-III plays a critical role in controlling triglyceride metabolism, and elevated levels of ApoC-III are associated with impaired triglyceride breakdown. By reducing ApoC-III, Waylivra helps lower the concentration of triglycerides in the blood, thus reducing the risk of pancreatitis and other complications associated with FCS.

    Indications

    Waylivra is indicated for the following:
    • Treatment of Familial Chylomicronemia Syndrome (FCS): Waylivra is used in adults with familial chylomicronemia syndrome (FCS) who are at high risk of severe pancreatitis and have inadequate response to dietary and other standard treatments for lowering triglycerides.

    Side Effects

    Common side effects of Waylivra include:
    • Injection site reactions (pain, redness, swelling)
    • Low platelet counts (thrombocytopenia)
    • Abdominal pain
    • Nausea
    • Fatigue
    • Fever
    • Headache
    Serious side effects include:
    • Severe thrombocytopenia (very low platelet levels)
    • Hepatotoxicity (liver damage)
    • Serious allergic reactions

    Contraindications

    Waylivra is contraindicated in the following conditions:
    • Severe thrombocytopenia (low platelet counts)
    • Patients with a known hypersensitivity to Volanesorsen or any of its components
    • Pregnant or breastfeeding women, as safety in pregnancy and lactation has not been established
    • Severe liver impairment

    Price of Waylivra (Volanesorsen) in Different Countries

    Prices for Waylivra vary depending on the country and healthcare system. Below is a table showing the approximate price of Waylivra in different regions:
    Country Price (per injection) Source
    United States $25,000 per month GoodRx
    United Kingdom £20,000 per month (NHS reimbursement) NICE BNF
    Canada CAD 32,000 annually DrugBank
    Germany €22,000 per month Medikamentenpreis
    Australia AUD 35,000 per month PBS Australia

    Top 5 Global Brands of Rare Disease Treatments

    Waylivra is a specialized treatment for a rare condition, but there are other global brands that focus on treating rare diseases and conditions. Some of the top global brands include:
    • Waylivra (Volanesorsen) by Ionis Pharmaceuticals and Akcea Therapeutics
    • Kynamro (Mipomersen) by Genzyme (Sanofi)
    • Spinraza (Nusinersen) by Biogen for spinal muscular atrophy (SMA)
    • Soliris (Eculizumab) by Alexion Pharmaceuticals for paroxysmal nocturnal hemoglobinuria (PNH)
    • Orkambi (Lumacaftor/Ivacaftor) by Vertex Pharmaceuticals for cystic fibrosis (CF)
     
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