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
- 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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