Elosulfase alfa

What is Elosulfase alfa?

Elosulfase alfa (Vimizim) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme used for the treatment of Morquio syndrome which is caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase.

What are the uses of this medicine?

• This medicine is used for the treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

How does this medicine work?

• Vimizim is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S.
• Elosulfase alfa uptake by cells into lysosomes is mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of elosulfase alfa to mannose-6-phosphate receptors.
• Treatment of MPS IVA chondrocytes with elosulfase alfa induced clearance of KS lysosomal storage from the chondrocytes.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• No formal clinical drug interaction studies have been performed with Vimizim .

Is this medicine FDA approved?

• It was approved for use in the United States in 2014.

How should this medicine be used?

• Pre-treatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of the infusion.

Recommended Dosage:

• The recommended dose is 2 mg per kg given intravenously over a minimum range of 3.5 to 4.5 hours, based on infusion volume, once every week.

Administration

• Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit.
• This product should be prepared and administered under the supervision of a healthcare professional with the ability to manage medical emergencies.
• Determine the number of vials to be diluted based on the individual patient’s weight and the recommended dose of 2 mg/kg.
• Dilute the calculated dose to a final volume of 100 mL or 250 mL using 0.9% Sodium Chloride Injection, USP.
• Bullet list

The final volume is based on the patient’s weight as follows:

• For patients who weigh less than 25 kg, the final volume should be 100 mL;
• For patients who weigh 25 kg or more, the final volume should be 250 mL.

• The solution should be clear to slightly opalescent and colorless to pale yellow when diluted.
• Do not use if the solution is discolored or if there is particulate matter in the solution.
• Note that a diluted solution with slight flocculation (e.g., thin translucent fibers) is acceptable for administration.
• Avoid agitation during preparation.
• Gently rotate the bag to ensure proper distribution.
• Do not shake the solution.
• Administer the diluted solution to patients using a low-protein binding infusion set equipped with a low-protein binding 0.2 micrometer (µm) in-line filter.
• For patients who weigh less than 25 kg: initial infusion rate should be 3 mL per hour for the first 15 minutes and, if tolerated, increased to 6 mL per hour for the next 15 minutes.
• For patients who weigh 25 kg or more: initial infusion rate should be 6 mL per hour for the first 15 minutes and, if tolerated, the infusion rate may be increased to 12 mL per hour for the next 15 minutes.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form: As Injection: 5 mg/5 mL (1 mg/mL) in single-dose vials

This medicine is available in fallowing brand namesː

• Vimizim

What side effects can this medication cause?

The most common side effects of this medicine include:

• pyrexia
• vomiting
• headache
• nausea
• abdominal pain
• chills
• fatigue

What special precautions should I follow?

• Anaphylaxis and hypersensitivity reactions have been reported in patients treated with Vimizim. If anaphylaxis or severe hypersensitivity reactions occur, immediately stop the infusion and initiate appropriate medical treatment. Pre-treatment with antihistamines with or without antipyretics is recommended prior to the start of infusion.
• 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.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• Available data from published case reports and postmarketing experience with Vimizim use in pregnant women are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.
• There is a Morquio A Registry that collects data on pregnant women with MPS IVA who are treated with Vimizim. Contact MARS@bmrn.com or call 1-800-983-4587 for information and enrollment.

Can this medicine be used in children?

• Safety and effectiveness of Vimizim have been established in pediatric patients 5 years of age and older.
• Safety and effectiveness in pediatric patients below 5 years of age have not been established.

What are the active and inactive ingredients in this medicine?

Active Ingredient:

• ELOSULFASE ALFA

Inactive Ingredients:

Who manufactures and distributes this medicine?

• Packager: BioMarin Pharmaceutical Inc.

What should I know about storage and disposal of this medication?

• Store Vimizim under refrigeration at 2°C to 8°C (36°F to 46°F).
• Do not freeze or shake.
• Protect from light.
• Diluted Vimizim should be used immediately.
• If immediate use is not possible, diluted Vimizim may be stored for up to 24 hours at 2°C to 8°C (36°F to 46°F) followed by up to 24 hours at 23°C to 27°C (73°F to 81°F) during administration.

• Dailymed label info on Elosulfase alfa
• FDA Elosulfase alfa

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