Prevention & lipidsResearch article
Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy
- Single-arm trial
- open-label
- Phase 3
- Median (range) treatment duration 104.3 (28.3-196.3) weeks
Authors' conclusion
In this large cohort of patients with HoFH, evinacumab was generally well tolerated and markedly decreased LDL-C irrespective of age and sex. Moreover, the efficacy and safety of evinacumab was sustained over the long term.
Study context
- Population
- Patients aged ≥12 years with homozygous familial hypercholesterolaemia (HoFH)102 adults · 14 adolescents
- Participants
- 116 patients
- Outcome
- Long-term safety and efficacy of evinacumab
Main finding
As reported in the abstract
LDL-C change from baseline · Week 24
| Group | Value | Mean (SD) LDL-C decrease |
|---|---|---|
| Evinacumab | mean decrease 43.6% | 3.4 (3.2) mmol/L |
Further findings
| Outcome | Values |
|---|---|
| LDL-C change from baseline, by age (subgroup) · Week 24 | Adults mean reduction 41.7% · Mean (SD) LDL-C reduction 3.2 (3.3) mmol/L · Adolescents mean reduction 55.4% · Mean (SD) LDL-C reduction 4.7 (2.5) mmol/L |
| Deaths · neither considered related to evinacumab | Evinacumab 1.7% |
Safety
Treatment-emergent adverse events (TEAEs) (overall population)
- TEAEs
- 93 (80.2%)
- Serious TEAEs
- 27 (23.3%)
Original publication
Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy. Eur Heart J. Published 10 June 2024. doi:10.1093/eurheartj/ehae325
Summarised from the published abstract; every number is checked against it