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

Glossary

This is a collection of words or phrases that you may hear your doctor mention, or you may see during your research into Type 1 Gaucher disease.

a

Active-controlled

a known, effective drug or treatment that is compared with the drug or treatment being investigated

Adverse reaction

side effect or unwanted effect that is possibly related to a drug

Amino acid

the building blocks of proteins; the body links amino acids together in specific ways to build proteins

Anaphylaxis

a severe, potentially life-threatening allergic reaction

Anemia

low levels of red blood cells

Antibody

a protective protein produced by your immune system in response to foreign substances

b

Baseline

a predefined early time point of a clinical study used to compare changes against; this could be the start of the study

c

Carrier

an individual who “carries” and can pass on a gene associated with a disease or a trait that is inherited by their children

Clinical study/Clinical trial

a research study involving human volunteers that investigates a drug or treatment

Contraindication

a specific medical condition that is a reason for a person not to receive a particular drug or treatment, as it may be harmful

d

Double-blind

a type of clinical study in which participants are assigned to treatment groups, and neither the participants nor the investigators know which treatment they receive

Drug–drug interaction

a reaction that occurs between two or more drugs

e

Endpoint

a research objective of a clinical study; clinical studies will have a primary endpoint, which is the main research objective, and secondary endpoints

Enzyme

a complex protein that speeds up a specific chemical reaction

Enzyme replacement therapy (ERT)

a treatment that works by replacing the missing or deficient enzyme in a patient

Extension study

a scenario where participants in an initial study continue to take the study drug or treatment for a further period to continue the investigation

g

Gaucher (GO-SHAY) cells

normal cells that become full of unprocessed glucocerebroside and accumulate primarily in the spleen, liver, and bone marrow, causing organ inflammation and dysfunction

Gaucher disease

an inherited disorder that affects many of the body’s organs and tissues

Gene

a sequence of nucleotides that forms the basic structural unit of deoxyribonucleic acid (DNA); genes provide instructions to make molecules called proteins, and every person has two copies of each gene, one inherited from each parent

Genetic counselor

a healthcare professional who can guide and support patients seeking more information about how inherited diseases and conditions might affect them, and can help to explain what test results mean

Genetic screening

a medical test that can confirm or rule out a suspected genetic condition or help determine a person’s chance of developing or passing on a genetic disorder

Genetic testing

the study of a person’s DNA in order to identify genetic changes that could increase the risk of diseases

Geneticist

a doctor who provides comprehensive diagnostic, management, and genetic counseling services for patients with genetic disorders

Glucocerebrosidase (GLOO-ko-SER-e-bro-sy-daze)

an enzyme that helps break down a large molecule called glucocerebroside; people with Gaucher disease have insufficient levels of this enzyme

Glucocerebroside (GLOO-ko-SER-e-bro-side)

a fatty substance that accumulates in cells affected by Gaucher disease throughout the body

h

Hematologist

a specialist medical doctor with extra training in disorders related to blood, bone marrow, and the lymphatic system

Hemoglobin (HEE-moh-GLOH-bin)

a protein inside red blood cells that carries oxygen from the lungs to tissues and organs in the body and carries carbon dioxide back to the lungs

Hepatologist

a specialist medical doctor who diagnoses, treats, and manages problems associated with the liver, gallbladder, bile ducts, and pancreas

Hypersensitivity reaction

an extreme or unnecessary immune response that the body experiences in response to a toxic or foreign substance

i

Infusion(in-FYOO-zhun)

the injection of medication through a needle or catheter, usually intravenously into a vein

Infusion center

a site of care that focuses specifically on medicines delivered using infusions, usually intravenously

Internist

a medical doctor who specializes in the internal organs and systems of the body

Intravenous

taking place within or being administered into a vein

m

Mutation

a change in a DNA sequence that can be passed on to offspring

n

Neurologist

a medical doctor who diagnoses and treats problems related to the brain and nervous system

Noninferiority

a clinical study that investigates whether a new treatment is no worse than an active treatment that it is being compared with

o

Open-label

a type of clinical study in which participants and investigators both know which treatment participants are receiving

p

Placebo

an inactive substance or treatment that looks the same and is given in the same ways as an active drug or treatment being investigated

Platelet

blood cells that form in your bone marrow (a sponge-like tissue in your bones) and play a major role in blood clotting

Protein

molecules made up of amino acids that have various functions around the body

r

Randomized

a type of clinical study in which patients are randomly assigned to treatment groups

s

Single-arm

a type of clinical study in which all participants receive the same drug or treatment

important safety information <

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat.

important safety information <

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat. VPRIV should be administered under the supervision of a healthcare professional. Appropriate medical support should be available when VPRIV is administered.

Hypersensitivity reactions were the most commonly observed side effects in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, low blood pressure, high blood pressure, nausea, tiredness/weakness, and fever. 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 patients not previously treated, occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. After the drug was approved, additional hypersensitivity reactions of chest discomfort, difficulty breathing, itching and vomiting have been reported. In some cases, vomiting can be serious and require hospitalization and/or stopping the medication.

If anaphylactic or other acute reactions occur, seek immediate medical care. Your healthcare provider will immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment. A hypersensitivity reaction should be treated based on the severity of the reaction. Your healthcare provider may manage a reaction by slowing the infusion rate or treating with medicine such as antihistamines, fever-reducing agents and/or corticosteroids or possibly stopping the medication and then restarting with a longer infusion time. For patients who have had symptoms of hypersensitivity reaction to enzyme replacement therapy, the doctor may consider treating the patient with antihistamines and/or corticosteroids before an infusion to help prevent such a reaction from happening.

The most commonly reported side effects during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, increased time it takes for blood to clot, tiredness/weakness, and fever. In clinical studies, the overall frequency of side effects was generally higher in the patients not previously treated with ERT than in the patients who switched from imiglucerase to VPRIV.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

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. Side effects more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, increased time it takes for blood to clot, and fever.

The side effect profile in elderly patients was generally similar to that seen in pediatric and other adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering other existing medical conditions.

As with all therapeutic proteins, there is a potential for developing antibodies to VPRIV. In clinical studies, 1 of 54 (2%) patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. One additional patient developed antibodies to VPRIV during an extension study. It is unknown if having antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

For additional safety information, please click here for Full Prescribing Information including WARNING for Risk of Anaphylaxis, and discuss with your doctor
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

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