Vimizim 1 mg/ml Concentrate for solution for infusion

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Description

Vimizim (elosulfase alfa) is a medication used to treat mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome), a rare lysosomal storage disorder. Each vial contains 1 mg/ml of elosulfase alfa, which is administered as an intravenous infusion. Elosulfase alfa is an enzyme replacement therapy that provides the deficient enzyme N-acetylgalactosamine-6-sulfatase (GALNS) to patients with MPS IVA.

How It Works

Elosulfase alfa works by replacing the deficient GALNS enzyme in patients with MPS IVA. This enzyme is necessary for the breakdown of glycosaminoglycans (GAGs), which are complex sugar molecules. In individuals with MPS IVA, the lack of GALNS leads to the accumulation of GAGs in the cells, causing various symptoms and complications. By providing the missing enzyme, Vimizim helps reduce GAG accumulation, thereby alleviating the symptoms and preventing further damage.

Indications

  • Treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) in patients of all ages

Contraindications

  • Known hypersensitivity to elosulfase alfa or any of the excipients in the formulation
  • Patients with acute severe illness, particularly respiratory illness, should not receive Vimizim until the illness is under control

Side Effects

  • Infusion-related reactions (fever, chills, rash, hives, headache)
  • Nausea
  • Vomiting
  • Abdominal pain
  • Diarrhea
  • Fatigue
  • Upper respiratory tract infections
  • Pain in the extremities

Price in Different Countries

Country Price (per vial) References
United States $5,000 GoodRx Vimizim Price
United Kingdom £4,000 NHS Vimizim Price
Canada $6,000 Canada Drugs Vimizim Price
Australia $5,500 PBS Vimizim Price
India ₹400,000 1mg Vimizim Price

Top 5 Global Brands

  • Vimizim (BioMarin Pharmaceutical): The primary brand of elosulfase alfa used for the treatment of MPS IVA.
  • Aldurazyme (Sanofi Genzyme): Used for enzyme replacement therapy in patients with MPS I (Hurler syndrome).
  • Elaprase (Shire Pharmaceuticals): An enzyme replacement therapy for the treatment of MPS II (Hunter syndrome).
  • Naglazyme (BioMarin Pharmaceutical): Used for enzyme replacement therapy in patients with MPS VI (Maroteaux-Lamy syndrome).
  • Brineura (BioMarin Pharmaceutical): Used to treat CLN2 disease, a form of Batten disease.
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