Taliglucerase alfa

What is Taliglucerase Alfa?

• Taliglucerase Alfa (ELELYSO) is a hydrolytic lysosomal glucocerebroside-specific enzyme used to treat Gaucher's disease.

What are the uses of this medicine?

• This medicine is used for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease.

How does this medicine work?

• ELELYSO, a long term enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside.
• ELELYSO uptake into cellular lysosomes is mediated by binding of ELELYSO mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• No formal drug interaction studies have been conducted with ELELYSO.

Is this medicine FDA approved?

• Taliglucerase was approved for use as enzyme replacement therapy for type 1 Gaucher disease in the United States in 2011.

How should this medicine be used?

Recommended dosage:

Treatment-naïve patients:

• The recommended dosage of ELELYSO for long-term treatment is 60 units/kg of body weight administered every other week as a 60 to 120 minute intravenous infusion.

Patients switching from imiglucerase:

• Begin ELELYSO at the same unit/kg dose as the patient's previous imiglucerase dose.
• Administer ELELYSO every other week as a 60 to 120 minute intravenous infusion.
• Dosage adjustments can be based on achievement and maintenance of each patient's therapeutic goals.

Administration: Reconstitute, dilute and administer under the supervision of a healthcare professional. After reconstitution and dilution, the preparation should be administered via intravenous infusion and filtered through an in-line low protein-binding 0.2 μm filter. For pediatric patients:

• An initial infusion rate of 1 mL/minute should be used.
• After tolerability to ELELYSO is established, the infusion rate may be increased, but should not exceed the maximum recommended infusion rate of 2 mL/minute. The total volume of the infusion should be delivered over a minimum of 60 minutes.

For adult patients:

• An initial infusion rate of 1.2 mL/minute should be used.
• After tolerability to ELELYSO is established, the infusion rate may be increased, but should not exceed the maximum recommended infusion rate of 2.2 mL/minute.
• The total volume of the infusion should be delivered over a minimum of 60 minutes.
• As ELELYSO contains no preservative, the product should be used immediately once reconstituted.

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

This medicine is available in fallowing doasage form:

• As injection: 200 units lyophilized powder in a single-dose vial for reconstitution

This medicine is available in fallowing brand namesː

• ELELYSO

What side effects can this medication cause?

The most common side effects of this medicine include: In Treatment-Naïve Adults:

• headache
• arthralgia
• fatigue
• nausea
• dizziness
• abdominal pain
• pruritus
• flushing
• vomiting
• urticaria

In Patients Switched from Imiglucerase, after 9 Months on Treatment:

• arthralgia
• headache
• pain in extremity

What special precautions should I follow?

• Serious hypersensitivity reactions, including anaphylaxis, have occurred in some patients treated with ELELYSO. Observe patients during and after the infusion; immediately discontinue infusion if anaphylaxis occurs and initiate appropriate treatment. Reduction in the infusion rate and/or pre-medication may prevent subsequent reactions.

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?

• The limited available data on ELELYSO use in pregnant women are not sufficient to inform a drug-associated risk.

Can this medicine be used in children?

• The use of ELELYSO for treatment of pediatric patients with Type 1 Gaucher disease is supported by evidence of effectiveness from adequate and well-controlled trials of ELELYSO in adults.

What are the active and inactive ingredients in this medicine?

Active Ingredient:

• TALIGLUCERASE ALFA

Inactive Ingredients:

• MANNITOL
• POLYSORBATE 80
• SODIUM CITRATE, UNSPECIFIED FORM

Who manufactures and distributes this medicine?

Manufactured by

• Pfizer Inc.

NY

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

• Store ELELYSO refrigerated at 2° C to 8° C (36° F to 46° F) in the original carton to protect from light.
• Do not freeze.

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enzyme replacement therapy

• gaucher disease agents
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  • imiglucerase, taliglucerase alfa, velaglucerase alfa

glucosylceramide synthase inhibitors (substrate restriction therapy)

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lysosomal acid lipase deficiency agents

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Urea Cycle Disorder Agents

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Hematologic Agents

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• Dailymed label info on Taliglucerase alfa
• FDA Taliglucerase alfa

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