"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 Trials Experience
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.
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 enzyme replacement therapy (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 1. The most common adverse reactions were hypersensitivity reactions.
Table 1: Adverse Reactions Observed in ≥ 10% of
Adult and Pediatric Patients with Type 1 Gaucher Disease
Treated with VPRIV in the Pooled 5 Clinical Studies
|Naive to ERT N = 54||Switched from imiglucerase to VPRIV N = 40|
|Number of Patients (% )|
|Hypersensitivity reaction*||28 (52)||9 (23)|
|Headache||19 (35)||12 (30)|
|Dizziness||12 (22)||3 (8)|
|Pyrexia||12 (22)||5 (13)|
|Abdominal pain||10 (19)||6 (15)|
|Back pain||9 (17)||7 (18)|
|Joint pain (knee)||8 (15)||3 (8)|
|Asthenia/Fatigue||8 (15)||5 (13)|
|Activated partial thromboplastin time prolonged||6 (11)||2 (5)|
|Nausea||3 (6)||4 (10)|
|*Denotes any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis.|
Less common adverse reactions affecting more than one patient ( > 2% in the treatment-na´ve group and > 3% in patients switched from imiglucerase to VPRIV treatment) were bone pain, tachycardia, rash, urticaria, flushing, hypertension, and hypotension.
Adverse Reactions in Pediatric Patients
The safety profile of VPR IV was similar between pediatric patients (ages 4 to 17 years) and adult patients. Adverse reactions more commonly seen in pediatric patients compared to adult patients include ( > 10% difference): rash, aPTT prolonged, and pyrexia.
As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 enzyme 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 hypersensitivity 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 to VPRIV.
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 information provided.
Last reviewed on RxList: 5/21/2015
This monograph has been modified to include the generic and brand name in many instances.
Additional VPRIV Information
Report Problems to the Food and Drug Administration
Find out what women really need.