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Elosulfase Alfa

Medically reviewed by Last updated on Aug 14, 2020.


(el oh SUL fase AL fa)

Index Terms

  • Elosulfase alfa
  • N-acetylgalactosamine-6-sulfatase

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]

Brand Names: U.S.

  • Vimizim

Pharmacologic Category

  • Enzyme


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.

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

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.


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

Hypersensitivity: Hypersensitivity reaction (19%)

Immunologic: Antibody development (100%; neutralizing: 100%)

Nervous system: Headache (26%)

Miscellaneous: Fever (33%)

1% to 10%:

Hypersensitivity: Anaphylaxis (8%)

Nervous system: Chills (10%), fatigue (10%)

ALERT: U.S. 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.


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 C 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).

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Headache

• Loss of strength and energy

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• 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 an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, 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 limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.