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.
In clinical trials, 139 patients with Fabry disease (79 females, 60 males, 92% Caucasian, ages 16 to 72 years), who were naive to GALAFOLD or previously treated with enzyme replacement therapy, were exposed to at least one dose of GALAFOLD. Of the 139 patients, 127 patients were exposed to GALAFOLD 123 mg every other day for 6 months and 123 patients were exposed for greater than one year. The clinical trials included one randomized, double-blind, placebo-controlled clinical trial of 6 months duration followed by a 6-month open-label treatment phase (Study 1). A second trial was a randomized, open-label, active-controlled clinical trial of 18 months duration in patients with Fabry disease receiving enzyme replacement therapy who were randomized to either switch to GALAFOLD or continue enzyme replacement therapy (Study 2; NCT01218659). In addition, there were two open-label, long-term extension trials.
The most common adverse reactions reported with GALAFOLD (≥ 10%) during the 6-month placebo-controlled, double-blind phase of Study 1 were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia.
Table 1 shows adverse reactions reported in at least 5% of patients treated with GALAFOLD (and at a higher rate than placebo) during the 6-month placebo-controlled, double-blind phase of Study 1.
Table 1: Adverse Reactions* Reported During the First 6 Months of Treatment in Patients with Fabry Disease in Study 1
|Adverse Reaction||GALAFOLD %
(N = 34)
(N = 33)
|Urinary tract infection* *||15%||0|
|* reported in at least 5% of patients treated with GALAFOLD and at a higher rate than placebo
** included urinary tract infection, cystitis, and kidney infection
Adverse reactions reported in > 5% of patients who received GALAFOLD in the 6-month open-label treatment phase of Study 1, in Study 2, and in the long-term extension trials (N = 115, mean duration of treatment 2.7 years) included those reported in Table 1 with the addition of vomiting.
SRC: NLM .