elosulfase alfa

REMS

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

Contraind./Precautions ⬆ ⬇

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).

Adv. Reactions/Side Effects ⬆ ⬇

GI: abdominal pain, nausea, vomiting

Neuro: fatigue, headache

Misc: chills, fever, HYPERSENSITIVITY REACTIONS ANAPHYLAXIS(INCLUDING ANAPHYLAXIS)

Interactions ⬆ ⬇

Drug-drug:

None noted.

Availability ⬆ ⬇

Solution for injection : 1 mg/mL

Route/Dosage ⬆ ⬇

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

US Brand Names ⬆ ⬇

Action ⬆ ⬇

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.

Therapeutic effects:

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

Classifications ⬆ ⬇

Therapeutic Classification: orphan drugs

Pharmacologic Classification: enzymes

Pharmacokinetics ⬆ ⬇

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).

Time/Action Profile ⬆ ⬇

(blood levels)

ROUTE	ONSET	PEAK	DURATION
IV	unknown‡	end of infusion	unknown

‡Improvement in musculoskeletal function may take wk-mos.

Patient/Family Teaching ⬆ ⬇

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.

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Indications ⬇