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  • Zokinvy (lonafarnib) 50mg Capsule

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    Description

    Zokinvy, with the active ingredient Lonafarnib, is an oral medication used for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS) and certain processing-deficient Progeroid Laminopathies. These are rare genetic disorders that cause premature aging in children. Zokinvy works by inhibiting the accumulation of defective progerin or progerin-like proteins that are implicated in the accelerated aging process in progeria. This treatment helps to reduce the symptoms and extend the lifespan of individuals affected by these conditions.

    How Zokinvy (Lonafarnib) Works

    Zokinvy (Lonafarnib) is a farnesyltransferase inhibitor (FTI). Farnesyltransferase is an enzyme involved in the post-translational modification of proteins like prelamin A, which is converted into progerin in patients with progeria. Progerin accumulates abnormally in the cells of individuals with HGPS, disrupting the structural integrity of the cell nucleus and accelerating aging processes. By inhibiting farnesyltransferase, Zokinvy prevents the farnesylation (attachment of a farnesyl group) of progerin, reducing its accumulation and the harmful effects it has on cellular functions, thereby slowing down disease progression.

    Indications

    Zokinvy is indicated for the following:
    • Hutchinson-Gilford Progeria Syndrome (HGPS): Zokinvy is used to reduce the risk of mortality in children diagnosed with this rare genetic disorder that causes premature aging.
    • Processing-Deficient Progeroid Laminopathies: It is also indicated for certain progeroid laminopathies where genetic mutations lead to defective lamin A proteins.

    Side Effects

    Common side effects of Zokinvy include:
    • Nausea
    • Vomiting
    • Diarrhea
    • Fatigue
    • Abdominal pain
    • Appetite loss
    • Headache
    More serious side effects include:
    • Liver enzyme elevations (hepatotoxicity)
    • QT prolongation (heart rhythm disturbance)
    • Dehydration (due to severe diarrhea)
    • Electrolyte imbalances

    Contraindications

    Zokinvy is contraindicated in the following conditions:
    • Hypersensitivity to Lonafarnib or any of the product’s excipients
    • Use with strong or moderate CYP3A inhibitors (as this may increase the risk of adverse reactions)
    • Severe hepatic impairment (due to risk of worsening liver function)
    • Pregnancy and breastfeeding (as safety has not been established in these populations)
    • Patients with QT prolongation or taking medications that can prolong the QT interval

    Price of Zokinvy (Lonafarnib) in Different Countries

    The price of Zokinvy varies significantly based on the region and healthcare system. Below is a table showing the approximate cost of Zokinvy in different countries:
    Country Price (per month) Source
    United States $23,000 per month GoodRx
    United Kingdom £18,500 per month (NHS) NICE BNF
    Germany €20,000 per month Medikamentenpreis
    Canada CAD 30,000 per month DrugBank
    Australia AUD 33,000 per month PBS Australia

    Top 5 Global Brands for Rare Genetic Disease Treatments

    While Zokinvy is one of the leading treatments for rare genetic diseases like progeria, there are other major global brands that specialize in rare diseases. These include:
    • Zokinvy (Lonafarnib) by Eiger BioPharmaceuticals
    • Kalydeco (Ivacaftor) by Vertex Pharmaceuticals for cystic fibrosis
    • Spinraza (Nusinersen) by Biogen for spinal muscular atrophy (SMA)
    • Brineura (Cerliponase alfa) by BioMarin for Batten disease
    • Soliris (Eculizumab) by Alexion Pharmaceuticals for paroxysmal nocturnal hemoglobinuria (PNH)
     
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    Zokinvy (lonafarnib) 50mg Capsule
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