Evinacumab plus standard care estimated to add up to 12 years of life in those with HoFH
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Key takeaways:
- A modeling analysis estimated evinacumab plus standard-of-care therapy increased survival in patients with genetic high cholesterol.
- Addition of evinacumab could increase life expectancy up to 12 years in HoFH.
Evinacumab-dgnb was estimated to increase life expectancy an additional 12 years among patients with homozygous familial hypercholesterolemia already on standard-of-care lipid-lowering therapies, researchers reported.
Addition of the angiopoietin-like 3 protein inhibitor evinacumab-dgnb (Evkeeza, Regeneron) on top of high-intensity statin therapy, PCSK9 inhibition and ezetimibe could increase average survival in patients with homozygous familial hypercholesterolemia (HoFH) from age 65 years up to 77 years, according to data from Regeneron Pharmaceuticals published in the European Journal of Preventive Cardiology.
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“Untreated, the average age of death can be as low as 18 years, with many patients with HoFH not expected to live into their third decade. Chances of survival depend mainly on the degree of serum LDL-C control, hence why treatment to reduce LDL-C levels as early and aggressively as possible is critical,” Jing Gu, senior manager of health economics and outcomes research at Regeneron, and colleagues wrote. “Due to the rarity of HoFH, it is not possible to determine the effect of lipid-lowering therapies on survival outcomes via clinical trials. In instances where disease prevalence is low, mathematical modeling approaches are often undertaken to ascertain the impact of treatment on survival. Thus, the primary objective of this research was to use a mathematical model to estimate the potential impact of evinacumab-dgnb on life expectancy in an HoFH population.”
Evinacumab-dgnb is currently approved by the FDA to treat HoFH in children as young as 5 years.
Model to estimate impact of evinacumab on life expectancy
To estimate the impact of evinacumab-dgnb on life expectancy of patients with HoFH, Gu and colleagues developed models using data from the phase 3 ELIPSE HoFH trial in addition to peer-reviewed efficacy data on other standard-of-care lipid-lowering therapies. The researchers compared high-intensity statin therapy; high-intensity statins plus ezetimibe; high-intensity statins plus ezetimibe and PCSK9 inhibition; high-intensity statins plus ezetimibe, PCSK9 inhibition and evinacumab-dgnb; and nontreatment strategies.
As Healio previously reported, the results of the ELIPSE HoFH trial showed an approximately 49% reduction in LDL among evinacumab-treated patients with HoFH, in addition to reductions in apolipoprotein B, non-HDL and total cholesterol compared with placebo.
The researchers reported that overall median survival among untreated patients with HoFH was age 33 to 43 years, depending on baseline LDL.
In the most robust model, Gu and colleagues estimated that high-intensity statin therapy increased survival by a median of 9 years and addition of ezetimibe increased survival another 9 years. Addition of PCSK9 inhibition further improved by median survival another 14 years, according to the study.
Moreover, the researchers estimated that the addition of evinacumab-dgnb on top of high-intensity statin therapy, ezetimibe and PCSK9 inhibition increased median survival by another 12 years, from age 65 to 77 years.
Real-world HoFH registries needed
“We estimated that adding evinacumab on top of standard-of-care lipid-lowering therapies ... substantially increased median survival of HoFH patients by 7 to 12 years, depending on assumptions on baseline LDL-C levels and hazard ratio for mortality due to LDL-C reduction. Therefore, the availability of evinacumab greatly helped to address unmet needs of HoFH population and improved their life expectancy to a level closer to the U.S. general population,” the researchers wrote. “Future research should focus on developing real-world registries for HoFH patients to facilitate the knowledge of this ultra-rare disease.”
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