• Treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA, Morquio A Syndrome).

Contraindicated in:

• None noted.

Use Cautiously in:

• Concurrent acute febrile/respiratory illness (↑ risk of life threatening complications of hypersensitivity reaction)
• Sleep apnea (supportive measures should be available during infusion)
• OB: Use during pregnancy only if potential maternal benefit justifies potential fetal risk
• Lactation: Use while breastfeeding only if potential maternal benefit justifies potential risk to infant
• Pedi: Children <5 yr (safety and effectiveness not established).

GI: abdominal pain, nausea, vomiting.

Neuro: fatigue, headache.
Misc: chills, fever, (INCLUDING ANAPHYLAXIS) HYPERSENSITIVITY REACTIONS ANAPHYLAXIS.

Drug-Drug:

• None noted.

• Solution for injection : 1 mg/mL;

• IV (Adults and Children ≥5 yr): 2 mg/kg once weekly.

Vimizim

• Consists of an enzyme produced by recombinant DNA technology that hydrolyzes sulfate from galactose-6–sulfate or N-acetyl-galactosamine-6–sulfate. Lack of this enzyme in Mucoplysaccharidosis results in intracellular accumulation of glycosaminoglycans (GAG) producing widespread cellular, tissue and organ dysfunction.

• Decreased accumulation of GAG, decreased end organ dysfunction and improved musculoskeletal performance MPS IVA.

Therapeutic Classification:

Pharmacologic Classification: enzymes

Absorption: IV administration results in complete bioavailability.

Distribution: Taken up by cells.

Metabolism/Excretion: Unknown.

Half-life: Initially: 7 min ; after prolonged treatment: 35.9 min (due to neutralizing antibodies).

(blood levels)

†Improvement in musculoskeletal function may take wk-mos.

• Explain the purpose of elosulfase alfa to patient.
• Advise patient to notify health care professional immediately if signs and symptoms of anaphylaxis occur.
• Advise female patients to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Encourage patient to enroll in Morquio A Registry to collect data on pregnant women and breastfeeding women with MPS IVA treated with Vimizim by contacting [email protected] or call 1-800-983-4587.

el-oh-SUL-fase AL-fa