"The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).
Clinical Studies Experience
The data described below reflect exposure of 94 patients with type 1 Gaucher disease who received VPRIV at doses ranging from 15 Units/kg to 60 Units/kg every other week in 5 clinical studies. Fifty-four (54) patients were na´ve to ERT and received VPRIV for 9 months and 40 patients switched from imiglucerase to VPRIV treatment and received VPRIV for 12 months [see Clinical Studies]. Patients were between 4 and 71 years old at time of first treatment with VPRIV, and included 46 male and 48 female patients.
The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions [see WARNINGS AND PRECAUTIONS].
The most commonly reported adverse reactions (occurring in ≥ 10% of patients) that were considered related to VPRIV are shown in Table 2. The most common adverse reactions were infusion-related reactions.
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
Table 2: Adverse Reactions Observed in ≥ 10% of
Patients with Type 1 Gaucher Disease Treated with VPRIV
|System Organ Class Preferred Term||Na´ve to ERT
N = 54
|Switched from imiglucerase to VPRIV
N = 40
|Number of Patients (% )|
|Nervous system disorders|
|Headache||19 (35)||12 (30)|
|Dizziness||12 (22)||3 (8)|
|Abdominal pain||10 (19)||6 (15)|
|Nausea||3 (6)||4 (10)|
|Musculoskeletal and connective tissue disorders|
|Back pain||9 (17)||7 (18)|
|Joint pain (knee)||8 (15)||3 (8)|
|Infections and infestations|
|Upper respiratory tract infection||17 (31)||12 (30)|
|Activated partial thromboplastin time prolonged||6 (11)||2 (5)|
|General disorders and administration site conditions|
|Infusion-related reaction*||28 (52)||9 (23)|
|Pyrexia||12 (22)||5 (13)|
|Asthenia/Fatigue||7 (13)||5 (13)|
|*Denotes any event considered related to and occurring within up to 24 hours of VPRIV infusion|
Less common adverse reactions affecting more than one patient ( > 3% in the treatment-na´ve group and > 2% in patients switched from imiglucerase to VPRIV treatment) were bone pain, tachycardia, rash, urticaria, flushing, hypertension, and hypotension.
All adult adverse reactions to VPRIV are considered relevant to pediatric patients (ages 4 to 17 years). Adverse reactions more commonly seen in pediatric patients compared to adult patients include ( > 10% difference): upper respiratory tract infection, rash, aPTT prolonged, and pyrexia.
As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 treatment-na´ve patients treated with VPRIV developed IgG class antibodies to VPRIV.
In this patient, the antibodies were determined to be neutralizing in an in vitro assay. No infusion-related reactions were reported for this patient. 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 enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies.
Immunogenicity assay results are highly dependent on the sensitivity and specificity of the assay. Additionally, the observed incidence of antibody positivity in an assay may be influenced by several factors, including assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease. For these reasons, comparison of the incidence of antibodies to VPRIV with the incidence of antibodies to other products may be misleading.
Read the VPRIV (velaglucerase alfa for injection) Side Effects Center for a complete guide to possible side effects
No drug-drug interaction studies have been conducted.
Last reviewed on RxList: 10/21/2013
This monograph has been modified to include the generic and brand name in many instances.
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