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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 to ELELYSO in 60 patients ages 13 to 74 years who received ELELYSO at doses ranging from 11 to 73 Units/kg every two weeks in 3 clinical studies, and included 31 males and 29 females. Thirty-two patients were naive to ERT (Study 1) and 28 were switched from imiglucerase to ELELYSO (Study 2) [see Clinical Studies]. Study 3 includes patients continuing treatment from Study 1 and Study 2. Twenty-four patients were treated for longer than 2 years and 4 patients were treated longer than 3 years.
Important adverse reactions including anaphylaxis, allergic reactions, and infusion reactions are described elsewhere in the label [see WARNINGS AND PRECAUTIONS]. One patient experienced a Type III immune-mediated skin reaction. The most common adverse reactions requiring interventions were infusion reactions [see WARNINGS AND PRECAUTIONS].
Table 2 is a listing of adverse reactions that occurred in 10% or greater of patients.
Table 2: Adverse Reactions that Occurred in ≥ 10% of Patients
Treated with ELELYSO
|Study 1||Study 2|
|Infusion reaction||14 (44%)||13 (46%)|
|URTI/Nasopharyngitis||1 (22%)||5 (18%)|
|Pharyngitis/Throat infection||6 (19%)||1 (4%)|
|Headache||6 (19%)||3 (11%)|
|Arthralgia||4 (13%)||3 (11%)|
|Influenza/Flu||4 (13%)||1 (4%)|
|UTI/Pyelonephritis||3 (9%)||3 (11%)|
|Back pain||1 (3%)||3 (11%)|
|Extremity pain||0||3 (11%)|
The types and incidences of adverse reactions with up to 24 months of treatment in study 3 were similar to study 1 and study 2.
In addition to those listed in Table 2, less commonly reported adverse reactions ( >2%) observed in clinical trials include fatigue, pain, pharyngolaryngeal pain, pruritus, diarrhea, dizziness, nausea, bone pain, abdominal pain, erythema, flushing, edema peripheral, muscle spasms, paresthesia, dyspnea, throat irritation, asthenia, chest discomfort, infusion site pain, alanine aminotransferase increased, musculoskeletal discomfort, musculoskeletal pain, insomnia, rash, and skin irritation.
As with all therapeutic proteins, patients have developed IgG anti-drug antibodies (ADA) to ELELYSO. In study 1, seventeen of 32 treatment naive patients (17/32,53%) who were administered ELELYSO every two weeks developed ADA post-treatment (defined as ADA positive at one or more post-treatment time points). Two additional patients were antibody positive at baseline; one patient withdrew after developing an allergic reaction with the first dose of ELELYSO and the second patient had increasing antibody titers with continued treatment. In study 2, four of 28 patients (4/28, 14%) switched from imiglucerase treatment to ELELYSO treatment once every two weeks developed ADA after the switch. One additional patient who switched from imiglucerase in Study 2 was positive at baseline but did not develop increased ADA titers after ELELYSO treatment. The relevance of ADA to therapeutic response and adverse events is currently unclear.
Using neutralizing antibody assays of limited sensitivity, two treatment naive patients (at 24 months of ELELYSO treatment) and one patient switched from imiglucerase (at 9 months of ELELYSO treatment) were determined to be positive for neutralizing activity in an in vitro enzyme inhibition assay and were negative in a cell based assay. For these patients there was no demonstrated association between positive neutralizing antibody assay results and therapeutic response. The significance of these findings is unknown at this time.
It is unknown if the presence of ADA to taliglucerase alfa is associated with a higher risk of infusion reactions. Patients who develop infusion or immune reactions with ELELYSO treatment should be monitored for ADA to ELELYSO. Additionally, patients with an immune reaction to other enzyme replacement therapies who are switching to ELELYSO should be monitored for ADA to ELELYSO.
Immunogenicity assay results are highly dependent on the sensitivity and specificity of the assay and may be influenced by several factors such as: assay methodology, sample handling, timing of sample collection, concomitant medication, and underlying disease. For these reasons, comparison of the incidence of antibodies to ELELYSO with the incidence of antibodies to other products may be misleading.
Last reviewed on RxList: 5/14/2012
This monograph has been modified to include the generic and brand name in many instances.
Additional Elelyso Information
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