Adult Dosing

Mucopolysaccharidosis type IVA (Morquio A Syndrome)

• 2 mg/kg IV qwk over a minimum range of 3.5-4.5 hours, based on infusion volume
• Pre-treatment with antihistamines with or without antipyretics is recommended 30-60 minutes before the start of the infusion

For patients 25 kg

• Dose should be diluted to a final volume of 250 mL using 0.9% Sodium Chloride Injection
• Initial infusion rate should be 6 mL/hr for first 15 mins, if tolerated may increase to 12 mL/hr for next 15 mins, further rate can be increased every 15 minutes in 12 mL/hr increments. Max: 72 mL/hr
• Total volume of the infusion should be delivered over a minimum of 4.5 hours

Note:

• Diluted solution must be administered using a low-protein binding infusion set equipped with a low-protein binding 0.2 micrometer (µm) in-line filter

Pediatric Dosing

Mucopolysaccharidosis type IVA (Morquio A Syndrome)

• Safety and effectiveness have not been established in pediatric patients <5 years of age
• 2 mg/kg IV qwk over a minimum range of 3.5-4.5 hours, based on infusion volume
• Pre-treatment with antihistamines with or without antipyretics is recommended 30-60 minutes before the start of the infusion

For patients <25 kg

• Dose should be diluted to a final volume of 100 mL using 0.9% Sodium Chloride Injection
• Initial infusion rate should be 3 mL/hr for first 15 mins, if tolerated may increase to 6 mL/hr for next 15 mins, further rate can be increased every 15 minutes in 6 mL/hr increments. Max: 36 mL/hr
• Total volume of the infusion should be delivered over a minimum of 3.5 hours

For patients 25 kg

• Dose should be diluted to a final volume of 250 mL using 0.9% Sodium Chloride Injection
• Initial infusion rate should be 6 mL/hr for first 15 mins, if tolerated may increase to 12 mL/hr for next 15 mins, further rate can be increased every 15 minutes in 12 mL/hr increments. Max: 72 mL/hr
• Total volume of the infusion should be delivered over a minimum of 4.5 hours

Note:

• Diluted solution must be administered using a low-protein binding infusion set equipped with a low-protein binding 0.2 micrometer (µm) in-line filter

• Treatment for patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome)

• Hypersensitivity to any of the ingredients of the product

• Life-threatening anaphylactic reactions presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms in conjunction with urticaria, have been reported to occur during infusions, regardless of the duration of the course of treatment
• Closely monitor patients during therapy and manage anaphylaxis. Advise patient about signs and symptoms of anaphylaxis after initiating therapy
• Additional monitoring is required in patients with acute respiratory illness as they are at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions

Renal Dose Adjustment

• Renal impairment: Dose adjustments not defined

Hepatic Dose Adjustment

• Hepatic impairment: Dose adjustments not defined

• Life-threatening anaphylaxis and hypersensitivity reactions have been reported in some patients during treatment. 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
• 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
• Spinal or cervical cord compression (SCC) is a serious complication of MPS IVA and may occur as part of the natural history of the disease. Patients with MPS IVA should be monitored for signs and symptoms of SCC and given appropriate treatment
• Use in Pediatric Patientes less than 5 years of age: Safety and effectiveness have not been established

Pregnancy Category:C

Breastfeeding: Manufacturer advises caution

• GI: Vomiting, nausea, abdominal pain
• CNS: Headache
• MISC: Pyrexia, chills, fatigue

• Enzymes; replaces deficient enzyme N-acetylgalactosamine-6 sulfatase (GALNS) to minimize progressive multisystemic manifestations of Morquio A syndrome
• Metabolism: Unknown
• Excretion: Unknown
• Half life: 7.5-35.9 mins

US Trade Name(s)

• Vimizim

US Availability

Vimizim

• INJ: 1 mg/ml (5 mL vial)

Canadian Trade Name(s)

• N/A

Canadian Availability

• N/A

UK Trade Name(s)

• N/A

UK Availability

• N/A

Australian Trade Name(s)

• N/A

Australian Availability

• N/A

Endocrine/Metabolic

Enzymes