An analysis of four clinical trials testing Cerdelga (eliglustat) for Gaucher disease patients shows the treatment is safe and without serious adverse events.
The study, “A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration,” was published in the journal Blood Cells, Molecules, and Diseases.
Therapy for Gaucher disease has relied on two approaches, enzyme replacement therapy (ERT) or oral substrate reduction therapy (SRT). While ERT mode of action works by decreasing the accumulation of glucosylceramide (GL-1), SRT’s approach is to slow down the production of GL-1.
There are currently two SRT drugs that are commercially available: Zavesca (miglustat), suitable for treating adults with mild or moderate Gaucher disease type 1 (GD1) for whom ERT is unsuitable; and Cerdelga, a more recent drug that is suitable as a first-line therapy for adults with GD1 and a compatible CYP2D6 metabolizer phenotype (CYP2D6 is a key enzyme involved in the metabolism of chemical substances).
However, “Clinicians have requested more information about frequency, timing, and duration of adverse events associated with eliglustat [Cerdelga],” researchers wrote.
The study’s authors analyzed adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral Cerdelga: the Phase 2 single-arm study (NCT00358150); the Phase 3 ENGAGE randomized, placebo-controlled trial (NCT00891202); the Phase 3 ENCORE randomized, imiglucerase-controlled trial (NCT00943111); and the Phase 3 EDGE randomized, double-blind trial of once-daily vs. twice-daily dosing (NCT01074944).
The authors focused on the 10 adverse events described in the Cerdelga U.S. Prescribing Information (USPI) and EU Summary of Product Characteristics (SmPC) and analyzed them in light of frequency, drug-relatedness, severity, seriousness, duration, and timing of onset.
The adverse events analyzed included headache, arthralgia, diarrhea, nausea, fatigue, flatulence, abdominal pain, upper abdominal pain, back pain, and extremity pain.
In 10% or more of patients they found the following effects: headache (17%); arthralgia (14%); nasopharyngitis (13%); diarrhea (10%); and dizziness (10%).
“No relationship was observed between incidence of adverse events and dose of [Cerdelga], CYP2D6 metabolizer status, or peak or trough eliglustat plasma levels,” the researchers wrote. “Overall, 79% of all adverse events were classified as ‘Recovered/Resolved’ at the time of data cut.”
“The majority of adverse events noted in the [Cerdelga] USPI and EU SmPC were occasional, non-serious, non-severe, transient, not drug-related, and did not lead to patients stopping the drug,” the study’s authors concluded.
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