Type 1 Gaucher disease is a rare, autosomal recessive, chronic, progressive disorder
caused by deficient activity of the lysosomal enzyme glucocerebrosidase1–3
Although rare, Gaucher disease is the most common type of lysosomal storage disease.3
Type 1 is the most common form of Gaucher, with a higher prevalence in the Ashkenazi Jewish population,
affecting1,3:
~1–9 IN 100,000
within the overall population4
~1 IN 600
within the Ashkenazi Jewish population5–7
~1 IN 17
within the Ashkenazi Jewish community that are carriers8
COMMON SIGNS & SYMPTOMS
OF TYPE 1 GAUCHER DISEASE
Gaucher disease is a multi-systemic and progressive disease.1,3 Symptom onset can
range from early childhood into late adulthood.9 Signs and symptoms may vary from patient to
patient.3 It is important to be aware of the possible combinations of signs and symptoms, as this can
help lead to earlier diagnosis.
LOW HEMOGLOBIN CONCENTRATION1
ANEMIA & FATIGUE1,3,10
LOW PLATELET COUNT1,3,10
BRUISING & BLEEDING1,3,10
ENLARGED SPLEEN1,3,10
ABDOMINAL DISTENSION & DISCOMFORT1,3,10
ENLARGED LIVER1,3,10
A combination of these signs and symptoms may be indicative of type 1 Gaucher disease. If you suspect type 1 Gaucher
disease, test now.
DIAGNOSTIC CHALLENGES
Accurate and timely diagnosis is important for the management of type 1 Gaucher disease.11
The diagnosis of type 1 Gaucher disease often occurs several years after the first onset
of clinical signs, and patients may experience diagnostic delays of up to 10 years.12,13 This
could be due to several factors:
As a rare disease, the overall awareness of Gaucher disease is limited13
The clinical manifestations are highly heterogeneous, both in type and severity,
presenting at a wide range of ages13,14
The earliest symptoms are general and non-specific so may be difficult to
recognize14
Patients may be misdiagnosed and referred to multiple specialists before receiving an
accurate diagnosis13,14
Delayed diagnosis can have serious medical consequences, including the development of potentially irreversible complications.13
TESTING FOR TYPE 1 GAUCHER DISEASE
Type 1 Gaucher disease can be confirmed with:
β-Glucosidase enzyme assay — the gold standard of diagnosing
type 1 Gaucher disease is a blood test that measures levels of glucosidase activity10,13
Summary of VPRIV, an ERT for type 1 Gaucher disease
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including anaphylaxis, have occurred. The most
serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions.
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.
IMPORTANT SAFETY INFORMATION
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
Hypersensitivity reactions, including anaphylaxis, have occurred. The most serious
adverse reactions in patients treated with VPRIV were hypersensitivity reactions.
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.
As with any intravenous protein product, hypersensitivity reactions are possible,
therefore, appropriate medical support, including personnel adequately trained in cardiopulmonary
resuscitative measures and access to emergency measures, should be readily available when VPRIV is administered. If
anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate
the appropriate medical treatment.
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. 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.
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.
Please click here
for Full Prescribing Information.
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.
For assistance with medical inquiries about VPRIV, please contact Takeda at
1-877-TAKEDA-7 (1-877-825-3327), or email medinfous@takeda.com.
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