Elosulfase alfa (Vimizim) is a once-weekly injectable medicine that is indicated for the treatment of Morquio A syndrome.

Morquio A syndrome is also called as mucopolysaccharidosis Type IV A that is a result of an enzyme deficiency (N-acetylgalactosamine-6-sulfatase or chondroitin sulfatase).

Elosulfase alfa (Vimizim) Uses:

• Mucopolysaccharidosis type IVA:

  • It is used to treat patients with Treatment of Morquio A syndrome (mucopolysaccharidosis type IVA, MPS IVA)

Elosulfase alfa (Vimizim) Dose in Adults

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

Elosulfase alfa (Vimizim) Dose in the treatment of Mucopolysaccharidosis type IVA (MPS IVA):

• 2 mg/kg intravenously once a week.

Elosulfase alfa (Vimizim) Dose in Childrens

Note:

• Premedicate 30 minutes to one hour before initiating the infusion with antihistamines with or without antipyretics.

Elosulfase alfa (Vimizim) Dose in the treatment of Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome):

• Children ≥5 years and Adolescents:

  • 2 mg/kg intravenously once a week.

• Canadian labeling:

  • Infants ≥9 months, Children, and Adolescents:
    • 2 mg/kg intravenously once a week.

Pregnancy Risk Factor C

• Animal reproduction studies have shown adverse outcomes for fetuses.
• It is also importat to note that mucopolysaccharidosis type IVA (MPS IVA) has the potential of causing adverse maternal and fetal outcomes.
• Pregnant women who use the drug to treat MPS IVA may register ([email protected], 1-800-983-4587).

Elosulfase alfa use during breastfeeding:

• It is unknown if the drug will be excreted into breastmilk.
• It is recommended that you use it with caution if you are breastfeeding.
• Breastfeeding mothers who use the drug in pregnancy to treat MPS IV could be included in the drug registry

Dose in Kidney disease: 

The manufacturer has not recommended any dose adjustment in patients with kidney disease.

Dose in Liver disease:

The manufacturer has not recommended any dose adjustment in patients with liver disease.

Common Side Effects of Elosulfase alfa (Vimizim):

• Central nervous system:

  • Headache

• Gastrointestinal:

  • Vomiting
  • Nausea
  • Abdominal pain

• Hypersensitivity:

  • Hypersensitivity reaction

• Miscellaneous:

  • Fever

Less Common Side Effects of Elosulfase alfa (Vimizim):

• Central nervous system:

  • Chills
  • Fatigue

• Hypersensitivity:

  • Anaphylaxis presenting as
    • Cough
    • Erythema
    • Throat tightness
    • Urticaria
    • Flushing
    • Cyanosis
    • Hypotension
    • Rash
    • Dyspnea
    • chest discomfort, and gastrointestinal symptoms

• Immunologic:

  • Immunogenicity

Contraindications to Elosulfase alfa (Vimizim):

There are no contraindications for its use, according to the manufacturer.

Warnings and precautions

• Formation of antibodies:

  • All patients treated developed antibodies to the drug during clinical trials.
  • It is unknown what effect these anti-drug antibody have on the efficacy or risk of infusion site reactions.

• Hypersensitivity/anaphylactoid reactions: [U.S. Boxed Warning]:

  • Serious allergic reactions can occur shortly after and during the infusion.
  • Anaphylactic reactions can be life-threatening and may include abdominal pain, shortness or chest pain, cough, wheezing or cyanosis.
  • They may also manifest as itching, flushing, flushing redness, low blood pressure, nausea, vomiting or tightening of the throat.
  • Patients must be closely monitored during infusions and should have access to appropriate medical support.
  • Patients suffering from respiratory disease or at risk for developing severe acute exacebations due to hypersensitivity reactions will need close observation.
  • Treatment must be stopped immediately if anaphylactic reactions occur.
  • Patients with a persistent or life-threatening reaction need to be monitored for a longer time.
  • Patients allergic to the drug should not be given the drug again.

• Infusion reactions

  • Infusion reactions can occur. They may appear as soon as the infusion is initiated, but they may not be apparent until six days later. Some reactions can be observed up to 47 days after infusion.
  • Before the infusion begins with antihistamines or antipyretics, patients should be premedicated
  • Infusion reactions can be treated by stopping treatment, decreasing the rate of infusion, or administering additional antipyretics. Corticosteroids might also be considered.
  • Stop the treatment immediately if you notice a severe reaction.

• Acute febrile illness and respiratory disease:

  • Acute febrile illness may require that treatment be delayed due to severe allergic reactions and anaphylactic reactions.

• Sleep Apnea

  • Patients with obstructive sleeping apnea should be cautious.
  • Antihistamines can increase the likelihood of sleep apnea.
  • If the patient experiences dyspnea, supplemental oxygen and CPAP (continuous negative airway pressure) should always be available.

• Compression of the cervical cord and spinal cord:

  • Patients may experience cord compression as a result of the disease process.
  • Monitor patients for signs and symptoms of spinal cord compression, such as back pain, limb paralysis, and urinary incontinence.

Monitoring parameters:

Monitor for infusion-related allergic reactions, and the clinical features of spinal cord compression.

How to administer Elosulfase alfa (Vimizim)?

IV:

• Before administering the drug, premedicate the patient with antihistamine with or without antipyretics (at least 30 - 60 minutes prior to the infusion).
• Administer the drug using an infusion set with low protein binding properties with in-line low protein-binding 0.2 micrometer filter.

Patients <25 kg:

• Infuse 100 mL solution @ 3 mL/hour for the first fifteen minutes.
• If no infusion-related reactions are noted and the infusion is well-tolerated, increase the rate of infusion to 6 mL/hour for the next 15 minutes.
• The infusion rate may be further increased in increments of 6 ml/minute every fifteen minutes if the drug is well-tolerated.
• The maximum rate of infusion is 36 mL/hour.
• The total volume of the infusion should be infused over 3.5 hours or more.

Patients ≥25 kg:

• Infuse 250 mL solution @ 6 mL/hour for the first fifteen minutes.
• If no infusion-related reactions are noted and the infusion is well-tolerated, increase the rate of infusion to 12 mL/hour for the next 15 minutes.
• The infusion rate may be further increased in increments of 12 ml/minute every fifteen minutes if the drug is well-tolerated.
• The maximum rate of infusion is 72 mL/hour.
• The total volume of the infusion should be infused over 4.5 hours or more.
• If a hypersensitivity reaction is noted the infusion may be infused at a slow rate, stopped temporarily, or discontinued.
• In case of a severe reaction, the infusion must be discontinued.
• The drug should not be infused with other products in the infusion tubing.
• After the drug is diluted, it must be delivered with in 48 hours of the dilution.

Mechanism of action of Elosulfase alfa (Vimizim):

• Elosulfase alfa is a recombinant form of the enzyme N-acetylgalactosamine-6-sulfatase that is produced in Chinese hamster cells.
• A deficiency of N-acetylgalactosamine-6-sulfatase result in the accumulation of the glycosaminoglycan (GAG) substrates (keratan sulfate and chondroitin-6-sulfate) in tissues. 
• This can lead to organ, tissue, and cellular dysfunction.
• Elosulfase alfa provides the exogenous recombinant form of the enzyme (N-acetylgalactosamine-6-sulfatase) that is taken into the lysosomes. 
• It increases the catabolism of the GAG substrates (such as keratan sulfate and chondroitin-6-sulfate).

Half-life elimination:

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

Time to peak:

• Week 0: 172 minutes;
• Week 22: 202 minutes

International Brand Names of Elosulfase alfa:

• Vimizim

Elosulfase alfa Brand Names in Pakistan:

No Brands Available in Pakistan.