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What is Type 1 Gaucher Disease?

Overview of Type 1 Gaucher Disease

Type 1 Gaucher disease is an inherited genetic disorder. In Gaucher disease, an enzyme called glucocerebrosidase (gloo-ko-ser-uh- bro-SI-dase) is deficient. This enzyme’s job is to break down a fatty substance called glucocerebroside (gloo-ko-SER-uh-bro-side).

When this enzyme doesn’t work properly, glucocerebroside builds up in the cells. These enlarged cells accumulate in the organs (particularly the spleen and liver) and tissues. This accumulation makes the organs unable to function properly and causes the signs of Gaucher disease.

Gaucher disease affects both males and females equally. It is the most common genetic disease in Ashkenazi (eastern and central European) Jewish people.

Common Signs of Type 1 Gaucher disease

  • Enlarged spleen and/or liver
  • Anemia
  • Low platelet count

Individuals who are affected may not show signs until well into adulthood, and severity of disease can vary a great deal.

In VPRIV clinical trials, specific laboratory measurements (hemoglobin concentration, platelet count, and liver and spleen volume) were evaluated and not general symptoms of type 1 Gaucher disease.

Incidence of Type 1 Gaucher Disease1,2


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Heredity

Type 1 Gaucher disease has an autosomal recessive inheritance pattern, so carriers do not have Gaucher disease but can pass it along to their biological children.

If both parents are carriers:

  • There is a 50% chance of being a carrier
  • There is a 25% chance of having the disease

Estimated Incidence1,2

  • 1 in 50,000 to 1 in 100,000 in the general population
  • 1 in 855 in the Ashkenazi Jewish population

References:
1.
National Library of Medicine. National Institutes of Health. Gaucher disease. http://www.nlm.nih.gov/medlineplus/ency/article/000564.htm. Accessed May 18, 2015.
2. GuggenbuhlP, GrosboisB, ChalèsG. Gaucher disease. Joint Bone Spine. 2008;75(2):116-124.

INDICATION

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

IMPORTANT SAFETY INFORMATION

  • Hypersensitivity reactions, including serious allergic reactions (anaphylaxis) have occurred. VPRIV (velaglucerase alfa for injection) should be administered under the supervision of a healthcare professional. VPRIV is given every other week by intravenous infusion that typically takes up to 60 minutes. Appropriate medical support should be available when VPRIV is administered. The most serious side effects in patients treated with VPRIV were hypersensitivity reactions.
  • 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.
  • If anaphylactic or other acute reactions occur, 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.
  • VPRIV is classified as pregnancy category B which means that animal reproduction studies did not show a risk to the unborn and there are no adequate and well-controlled studies in pregnant women. Your doctor may prescribe VPRIV to you if you are pregnant, only if it is clearly necessary.
  • 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 patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. 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.
  • Please see full prescribing information.
  • 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.