Velaglucerase alfa

What is Velaglucerase alfa?

• Velaglucerase alfa (VPRIV) is a hydrolytic lysosomal glucocerebroside-specific enzyme used as a long-term enzyme replacement therapy for those suffering of Gaucher disease Type 1.

What are the uses of this medicine?

• Velaglucerase alfa (VPRIV) is used for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

How does this medicine work?

• Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.

In clinical trials VPRIV reduced spleen and liver size, and improved anemia and thrombocytopenia.

• Velaglucerase is produced in a human cell line using gene activation technology, resulting in production of a recombinant protein with the identical amino acid sequence as the native, human enzyme.
• The glucocerebrosidase producing cells are treated with enzymes that modify glycosylation and result in high-mannose type glycans which increase uptake of the velaglucerase by macrophages.

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 VPRIV.

Is this medicine FDA approved?

• Velaglucerase was approved for use as enzyme replacement therapy of type 1 [[[Gaucher disease]] in the United States in 2010.
• It is available as a lyophilized powder in single use vials of 400 Units under the brand name Vpriv. The typical initial dose is 60 Units/kg intravenously every 2 weeks.

How should this medicine be used?

Recommended dosage: Recommended Starting Dose in Adults and Pediatric Patients 4 Years of Age or Older:

• Patients Naïve to Enzyme Replacement Therapy: 60 Units/kg
• Patients being treated with stable imiglucerase dosages for Gaucher disease: Can switch to VPRIV at previous imiglucerase dose two weeks after last imiglucerase dose
• Consider pre-treatment with antihistamines and/or corticosteroids in patients who exhibited symptoms of hypersensitivity associated with prior velaglucerase alfa product infusions.

Administration:

• Determine number of vials to be reconstituted based on patient's actual weight and prescribed dose.
• Supplied VPRIV lyophilized powder must be reconstituted with Sterile Water for Injection.
• Reconstituted VPRIV solution must be diluted in 100 mL of 0.9% Sodium Chloride Injection prior to intravenous infusion.
• Administer the diluted VPRIV solution through an in-line low protein-binding 0.2 or 0.22 µm filter over 60 minutes.
• Do not infuse VPRIV with other products in the same infusion tubing because the compatibility of a VPRIV solution with other products has not been evaluated.

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

This medicine is available in fallowing doasage form:

• As injection: 400 units lyophilized powder single-dose vials

This medicine is available in fallowing brand namesː

• VPRIV

What side effects can this medication cause?

The most common side effects of this medicine include:

• hypersensitivity reactions
• headache
• dizziness
• abdominal pain
• nausea
• back pain
• joint pain
• prolonged activated PTT
• fatigue/asthenia
• pyrexia

What special precautions should I follow?

• Hypersensitivity reactions, including anaphylaxis, occurred in clinical studies and postmarketing experience. In cases where patients have exhibited symptoms of hypersensitivity to velaglucerase alfa or excipients in the drug product or to other enzyme replacement therapy, pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions.
• Ensure that personnel administering product are adequately trained in cardiopulmonary resuscitative measures, and have ready access to emergency medical services.
• Consider slowing infusion rate, treatment with antihistamines, antipyretics and/or corticosteroids, and/or stopping treatment if a hypersensitivity reaction occurs during an infusion. Consider pre-treatment with antihistamines and/or corticosteroids in patients with prior 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?

• While available data cannot definitively establish or exclude the absence of a velaglucerase alfa associated risk during pregnancy, these data have not identified an association with use of velaglucerase alfa during pregnancy and major birth defects, miscarriage, or adverse maternal or fetal outcomes.

Can this medicine be used in children?

• The safety and effectiveness of VPRIV have been established for enzyme replacement therapy (ERT) in patients between 4 and 17 years of age with type 1 Gaucher disease.
• The efficacy and safety of VPRIV has not been established in pediatric patients younger than 4 years of age.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• VELAGLUCERASE ALFA

Inactive ingredients:

• CITRIC ACID MONOHYDRATE
• POLYSORBATE 20
• TRISODIUM CITRATE DIHYDRATE
• SUCROSE

Who manufactures and distributes this medicine?

• Manufactured by:

Takeda Pharmaceuticals U.S.A., Inc. Lexington, MA

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

• Store VPRIV refrigerated at 2ºC to 8ºC (36ºF to 46ºF) in the original carton to protect from light.
• Do not freeze.
• Do not use VPRIV after the expiration date on the vial.

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• Dailymed label info on Velaglucerase alfa
• FDA Velaglucerase alfa

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