Two-year postmarketing safety experience with oral eliglustat in adults with type 1 Gaucher disease

Hou, Audrey W.; Patel, Parija; Peterschmitt, Michel; Ibrahim, Johan Afendi; Gaemers, Sebastiaan J.M.

doi:10.1016/j.ymgme.2016.11.155

Cited by 2 publications

(1 citation statement)

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“…Thus, both clinical trial data and post-marketing data are important complementary components of the adverse event profile of any drug. Of note, an analysis of 2-years post-marketing “real world” adverse event data for eliglustat identified no new safety concerns and a similar favorable adverse event profile to this longer-term analysis as well as the earlier ISS analysis of clinical trial data; the most frequently reported adverse drug reactions were nausea, fatigue, dyspepsia, constipation, gastroesophageal reflux disease, and dizziness [37].…”

Section: Discussionmentioning

confidence: 93%

Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1

Peterschmitt

Freisens

Underhill

et al. 2019

Orphanet J Rare Dis

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Background Eliglustat is a first-line oral treatment for adults with Gaucher disease type 1 who have an extensive, intermediate or poor CYP2D6 metabolizer phenotype (> 90% of patients). Whereas enzyme replacement therapy for Gaucher disease has been widely used for more than two decades, eliglustat has only been in commercial use since 2014. Clinicians and patients want to better understand which adverse events are most commonly associated with eliglustat, as well as their severity, frequency, and duration. Methods This pooled analysis of treatment-emergent adverse events combines data from four completed eliglustat clinical trials involving 393 Gaucher disease type 1 patients. It represents 1400 patient-years of eliglustat exposure and a mean treatment duration of 3.6 years (maximum: 9.3 years). Results Eighty-one percent of patients remained in their respective trial until commercial availability of eliglustat (US patients only) or until trial completion. Nine patients (2.3%) withdrew from their respective trial due to one or more adverse events reported as eliglustat treatment-related; all but one of these events were mild or moderate. Overall, 97% of adverse events were mild or moderate and 86% were reported by the investigator as unrelated to eliglustat treatment. The overall rate of adverse events decreased over time and did not increase with increasing eliglustat dose. We evaluated frequency, duration, and severity of 14 adverse event terms reported at least once as treatment-related in 2% or more of all patients: dyspepsia (5.9%), headache (5.3%), abdominal pain upper (5.1%), dizziness (5.1%), diarrhea (4.6%), nausea (4.6%), arthralgia (3.6%), constipation (3.3%), abdominal pain (2.8%), gastroesophageal reflux disease (2.8%), fatigue (2.8%), palpitations (2.8%), abdominal distension (2.5%), and gastritis (2.3%). For abdominal pain upper, diarrhea, nausea, abdominal pain, and headache events, median duration was less than 14 days. All 14 adverse event terms, except for arthralgia and headache, were reported only once per patient in more than 70% of patients experiencing the event. Conclusions This final pooled analysis of treatment-emergent adverse events reinforces the favorable safety profile of eliglustat. The majority of the most frequently reported treatment-related adverse events were mild or moderate, transient, and occurred only once per patient. Electronic supplementary material The online version of this article (10.1186/s13023-019-1085-6) contains supplementary material, which is available to authorized users.

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Section: Discussionmentioning

confidence: 93%