Elosulfase Alfa

What it is and how it works

Mechanism of Action Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N -acetylgalactosamine-6-sulfatase activity. The sulfatase activity deficiency results in the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body. The accumulation leads to widespread cellular, tissue, and organ dysfunction.

Indications

(elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). VIMIZIM is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

Dosage and administration (summary)

Administration of VIMIZIM should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. 2 mg per kg body weight administered once every week as an intravenous infusion over a minimum of 3.5 to 4.5 hours, based on infusion volume. ( 2.2 , 2.4 ) See the full prescribing information for administration modifications due to hypersensitivity reactions. Important Administration Instructions Administration of VIMIZIM should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis .

Contraindications

None. None.

Warnings and precautions

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. Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with enzyme replacement therapies, including VIMIZIM.

Pregnancy

Pregnancy Risk Summary Available data from published case reports, a registry with a pregnancy sub-study and pharmacovigilance reports with VIMIZIM use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.

Storage and supply

Storage and Handling Store refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze or shake.

Source: Elosulfase Alfa (Vimizim) — US FDA prescribing information, 2025-10-30, DailyMed. Summarised and shortened; the full label governs. Iranian registration, indications and labelling may differ.