How VPRIV® Works | VPRIV® (velaglucerase alfa) for injection

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HOW ERT WORKS & VPRIV MOA

VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for first-line treatment for patients with type 1 Gaucher disease^1,2

HOW ERT WORKS

ERT replaces or supplements deficient glucocerebrosidase.^2,3

Glucocerebrosidase deficiency causes the build-up of lipids in the lysosomes, leading to the formation of Gaucher cells⁴
Over time, these Gaucher cells infiltrate various organs, particularly the spleen and liver, resulting in a progressive, heterogeneous disease⁴
ERT is an infusion that replaces or supplements the deficient glucocerebrosidase enzyme^2,3

ERT IS A FIRST-LINE TREATMENT

ERT is a first-line treatment for Gaucher disease that has been used for many years²
ERTs address the underlying enzyme deficiency^2,3
Treatment with ERTs has been used for visceral debulking (reducing spleen and liver volumes) and improving hemoglobin and platelet counts^2,3

VPRIV MECHANISM OF ACTION (MOA)

VPRIV, an ERT

VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term ERT for patients with type 1 Gaucher disease¹
VPRIV’s safety and efficacy were evaluated in the largest clinical trial program of an ERT for type 1 Gaucher disease; VPRIV was studied across three clinical trials (n=99; aged ≥4 years)^1,5,6

VPRIV is specifically designed to match and replace the natural human enzyme.^1,7

VPRIV is produced using gene-activation technology in a human cell line¹
VPRIV has the same amino acid sequence as the naturally occurring human enzyme, glucocerebrosidase¹

Glucocerebrosidase

VPRIV

VPRIV’s glycosylation pattern is intended to facilitate targeted uptake into macrophages.^*1,7–9

VPRIV Mechanism of Action Video

Gaucher cells are swollen due
to the accumulation of a fatty substance known as glucocerebroside.^10,11

VPRIV is designed to bind to, and be absorbed into, the cell. Once inside the cell, VPRIV breaks down glucocerebroside.¹

Like the naturally occurring human enzyme, VPRIV breaks down glucocerebroside, reducing the overall amount in the cell.¹

*For illustration purposes only. In vitro test results do not necessarily correlate with clinical efficacy

Summary of VPRIV, an ERT for type 1 Gaucher disease

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.

INDICATION

VPRIV® (velaglucerase alfa) for injection is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.

If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

Life-threatening hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including VPRIV. VPRIV-treated patients have had these reactions occur in clinical studies and postmarketing experience.

Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia. Hypersensitivity reactions in the clinical trials include any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis. Generally the reactions were mild and, in treatment-naïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. Additional hypersensitivity reactions of chest discomfort, dyspnea, pruritus and vomiting have been reported in post-marketing experience. In some cases vomiting can be serious, requiring hospitalization and/or drug discontinuation.

Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administration of VPRIV should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures including access to cardiopulmonary resuscitation equipment.

Management of hypersensitivity reactions should be based on severity of the reaction, such as slowing the infusion rate, treatment with medications such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. 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. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

The most common adverse reactions during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated partial thromboplastin time (aPTT), fatigue/asthenia, and pyrexia. In clinical studies, the overall frequency of adverse events was generally higher in the population naïve to enzyme replacement therapy (ERT) than in the population switched from imiglucerase to VPRIV.

The safety and efficacy profiles were similar in pediatric (ages 4 to 17) and adult patients. The safety of VPRIV has not been established in patients under 4 years of age. Adverse reactions more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, prolonged aPTT, and pyrexia. The adverse reaction profile in elderly patients was consistent with that previously observed across pediatric and adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering comorbid conditions.

As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 (2%) enzyme treatment-naïve patients treated with VPRIV developed IgG class antibodies (neutralizing in an in vitro assay). One additional patient developed IgG antibodies to VPRIV during an extension study. It is unknown if the presence of IgG antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other ERTs who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

To report SUSPECTED ADVERSE REACTIONS, contact Medical Information at 1-866-888-0660, option 2 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please click here for Full Prescribing Information, including Boxed WARNING for Risk of Anaphylaxis.

For more information, contact Takeda at 1-877-TAKEDA-7 (1-877-825-3327), or by email at medinfous@takeda.com