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Elosulfase Alfa

Generic name: elosulfase alfa systemic

Brand names: Vimizim

Boxed Warning

Risk of anaphylaxis:

Life-threatening anaphylactic reactions have occurred in some patients during elosulfase alfa infusions. Anaphylaxis, presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms (eg, nausea, abdominal pain, retching, vomiting) in conjunction with urticaria, have been reported to occur during elosulfase alfa infusions, regardless of duration of the course of treatment. Closely observe patients during and after elosulfase alfa administration and be prepared to manage anaphylaxis. Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur. Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring.

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Vimizim: 5 mg/5 mL (5 mL) [contains mouse (murine) and/or hamster protein]


Mechanism of Action

Elosulfase alfa is a recombinant form of N-acetylgalactosamine-6-sulfatase, produced in Chinese hamster cells. A deficiency of this enzyme leads to accumulation of the glycosaminoglycan (GAG) substrates (keratan sulfate and chondroitin-6-sulfate) in tissues, causing cellular, tissue and organ dysfunction. Elosulfase alfa provides the exogenous enzyme (N-acetylgalactosamine-6-sulfatase) that is taken into lysosomes and thereby increases the catabolism of the GAG substrates (eg, keratan sulfate and chondroitin-6-sulfate).



Vd: Week 0: 396 mL/kg; Week 22: 650 mL/kg

Time to Peak

Week 0: 172 minutes; Week 22: 202 minutes

Half-Life Elimination

Week 0: ~8 minutes; Week 22: ~36 minutes

Use: Labeled Indications

Mucopolysaccharidosis type IVA: Treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome)


There are no contraindications listed in the manufacturer's labeling.

Dosage and Administration

Dosing: Adult

Note: Premedicate with antihistamines with or without antipyretics 30 to 60 minutes prior to infusion.

Mucopolysaccharidosis type IVA (MPS IVA): IV: 2 mg/kg once weekly

Dosing: Pediatric

Note: Premedicate with antihistamines with or without antipyretics 30 to 60 minutes prior to infusion.

Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome):

Children ≥5 years and Adolescents: IV: 2 mg/kg once weekly

Canadian labeling: Infants ≥9 months, Children, and Adolescents: IV: 2 mg/kg once weekly


Dilute calculated dose in NS to a final volume of 100 mL (for patients weighing <25 kg) or 250 mL (for patients weighing ≥25 kg). Gently rotate to distribute. Do not shake or agitate. Use immediately. Vials are for single use only; discard any unused product.


IV: Administer using a low protein-binding infusion set with in-line low protein-binding 0.2 micrometer filter. Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to infusion.

Patients <25 kg: Infuse 100 mL solution at 3 mL/hour for the first 15 minutes. If well-tolerated, increase to 6 mL/hour for the next 15 minutes. If well-tolerated, increase rate every 15 minutes in 6 mL/hour increments; maximum infusion rate: 36 mL/hour. The total volume of the infusion should be delivered over ≥3.5 hours.

Patients ≥25 kg: Infuse 250 mL solution at 6 ml/hour for the first 15 minutes. If well-tolerated, increase to 12 mL/hour for the next 15 minutes. If well-tolerated, increase rate every 15 minutes in 12 mL/hour increments; maximum infusion rate: 72 mL/hour. The total volume of the infusion should be delivered over ≥4.5 hours.

The infusion can be slowed, temporarily stopped, or discontinued if a hypersensitivity reaction occurs. Discontinue immediately if severe reaction occurs. Do not infuse with other products in the infusion tubing. Administration should be completed within 48 hours from time of dilution.


Prior to use, store intact vials under refrigeration at 2°C to 8°C (36°F to 46°F); do not freeze or shake. Protect from light. Following dilution in NS, use immediately. If unable to use immediately, may store for up to 24 hours under refrigeration followed by up to 24 hours at 23°C to 27°C (73°F to 81°F) during administration.

Drug Interactions

There are no known significant interactions.

Adverse Reactions


Central nervous system: Headache (26%)

Gastrointestinal: Vomiting (31%), nausea (24%), abdominal pain (21%)

Hypersensitivity: Hypersensitivity reaction (19%)

Miscellaneous: Fever (33%)

1% to 10%:

Central nervous system: Chills (10%), fatigue (10%)

Hypersensitivity: Anaphylaxis (8%; presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms)

Immunologic: Immunogenicity


Concerns related to adverse effects:

  • Antibody formation: All patients developed anti-drug antibodies and neutralizing antibodies during clinical trials; it is unknown if presence of antibodies is related to a higher risk of infusion reactions or clinical efficacy treatment effect, respectively.
  • Hypersensitivity/anaphylactoid reactions: [U.S. Boxed Warning]: Serious hypersensitivity reactions, including life-threatening anaphylactic reactions have occurred, regardless of treatment course duration. Anaphylaxis may present as abdominal pain, chest discomfort, cough, cyanosis, dyspnea, erythema, flushing, hypotension, nausea, rash, retching, throat tightness, urticaria, and vomiting. Monitor closely during and after infusion. Appropriate medical support should be readily available. Patients with acute respiratory disease are at risk of serious acute exacerbation or respiratory compromise due to hypersensitivity; additional monitoring may be required. Discontinue immediately if anaphylactic or acute reaction occurs. Patients experiencing initial severe or refractory reactions may need prolonged monitoring. Use caution with readministration.
  • Infusion reactions: Infusion-related reactions have been reported; may be sporadic and/or severe. Hypersensitivity reactions may occur as early as 30 minutes from the start of infusion and have also been reported as late as 6 days after infusion; reactions may occur as late as the 47th infusion. Patients should be premedicated with antihistamines with or without antipyretics prior to infusion; evaluate airway prior to therapy (due to possible effects of antihistamine use). In case of reaction, decrease the rate of infusion, temporarily discontinue the infusion, and/or administer additional antipyretics/antihistamines and possibly corticosteroids. Discontinue treatment immediately if severe reaction occurs; use caution with readministration.

Disease-related concerns:

  • Acute febrile/respiratory illness: Consider delaying treatment in patients with an acute febrile or respiratory illness; may be at increased risk of life-threatening complications from hypersensitivity reactions.
  • Sleep apnea: Use with caution in patients with sleep apnea; antihistamine pretreatment may increase the risk of apneic episodes. Apnea treatment options (eg, supplemental oxygen or continuous positive airway pressure) should be readily available.
  • Spinal/cervical cord compression: Patients with MPA IVA may experience spinal/cervical cord compression (SCC) as a part of their disease. Monitor patients for signs and symptoms of SCC (eg, back pain, limb paralysis, urinary and fecal incontinence).

Monitoring Parameters

Monitor for infusion/hypersensitivity reactions; signs/symptoms of spinal or cervical compression.


Pregnancy Risk Factor


Pregnancy Considerations

Adverse events were observed in some animal reproduction studies. Mucopolysaccharidosis type IVA (MPS IVA) has the potential to cause adverse events in both the mother and fetus. A pregnancy registry is available for women who may be exposed to elosulfase alfa for the treatment of MPS IVA during pregnancy ( or 1-800-983-4587).

Patient Education

What is this drug used for?

  • It is used to treat mucopolysaccharidosis type IVA (MPS IVA or Morquio A syndrome).

Frequently reported side effects of this drug

  • Headache
  • Loss of strength and energy

Other side effects of this drug: Talk with your doctor right away if you have any of these signs of:

  • Infusion reaction like cough, skin redness, tightness in throat, flushing, blue/gray skin discoloration, severe dizziness, passing out, shortness of breath, chest pain, vomiting, or severe abdominal pain
  • Swelling of arms or legs
  • Cough
  • Back pain
  • Weakness, numbness, or tingling
  • Chills
  • Not able to move
  • Leaking of urine or stool
  • Signs of a significant reaction like wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a brief summary of general information about this medicine. It does NOT include all information about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not specific medical advice and does not replace information you receive from the healthcare provider. You must talk with the healthcare provider for complete information about the risks and benefits of using this medicine.

Source: Wolters Kluwer Health. Last updated November 21, 2019.