Vimizim

About Vimizim

• Vimizim is a prescription medicine first approved enzyme replacement therapy designed to address the underlying cause of Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). It is a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS) and Vimizim works at a cellular level to help with deficient enzyme activity.
• Morquio A is a rare and progressive inherited disease that targets the major organs in the body. Patients with Morquio A disease are missing an enzyme required for the breakdown and removal of the glycosaminoglycans (GAGs) called keratan sulfate (KS) and chondroitin-6-sulfate (C6S).

Strength: 

The dose of 2 mg per kg body weight is administered once every week as an intravenous infusion over a minimum of 3.5 to 4.5 hours.

Recommended Dosage:

• The recommended dose is 2 mg per kg given intravenously over a minimum range of 3.5 to 4.5 hours.
• Pre-treatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion.
• Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit.
• This product should be prepared and administered under the supervision of a healthcare professional with the ability to manage medical emergencies.
• Determine the number of vials to be diluted based on the individual patient’s weight and the recommended dose of 2 mg/kg.
• Dilute the calculated dose to a final volume of 100 mL or 250 mL using 0.9% Sodium Chloride Injection, USP.
• The solution should be clear to slightly opalescent and colorless to pale yellow when diluted. Do not use if the solution is discolored or if there is particulate matter in the solution. Note that a diluted solution with slight flocculation (e.g., thin translucent fibers) is acceptable for administration.
• Avoid agitation during preparation. Gently rotate the bag to ensure proper distribution. Do not shake the solution. 2.3 Administration Instructions Administer the diluted solution to patients using a low-protein binding infusion set equipped with a low protein binding 0.2 micrometer (µm) in-line filter.

Warnings & Precautions

• Anaphylaxis and Hypersensitivity Reactions: life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during treatment with Vimizim. 
• If anaphylaxis or severe hypersensitivity reactions occur, immediately stop the infusion and initiate appropriate medical treatment. 
• Pre-treatment with antihistamines with or without antipyretics is recommended prior to the start of infusion. 
• Risk of Acute Respiratory Complications: Patients with acute febrile or respiratory illness may be at higher risk of life-threatening complications from hypersensitivity reactions. 
• Careful consideration should be given to the patient’s clinical status prior to administration of Vimizim and consider delaying the Vimizim infusion.

Common Vimizim Side Effects

• Pyrexia
• Vomiting
• Headache
• Nausea
• Abdominal pain
• Chills
• Fatigue

Use in Specific Population

Pregnancy: It is a pregnancy category C. There are no adequate and well-controlled studies with Vimizim in pregnant women.

Pediatric use: The safety and effectiveness of Vimizim is established in pediatric patients 5 years of age and older. Use of Vimizim in patients 5 years of age and older is well supported by well-controlled trials in pediatric and adult patients. 

Storage and Handling

• The product should be used immediately after dilution as it contains preservatives.
• If the diluted product is not used immediately, it may be stored for up to 24 hours at 2°C to 8°C (36°F to 46°F) followed by up to 24 hours at 23°C to 27°C (73°F to 81°F). 
• Administration of Vimizim should be completed within 48 hours from the time of dilution of the product.
• Do not freeze or shake. Protect from light.
• Discard any unused product.