In the average person, you would expect to see normal to near-optimal low-density lipoprotein (LDL) cholesterol levels sitting at 129 mg/DL or lower; levels under 100 are seen as healthier. But for people with homozygous familial hypercholesterolemia (HoFH), a rare genetic form of high cholesterol, their LDL levels can rise to extremely high levels of 400 – 1,000 mg/DL. This can cause symptoms like yellowish waxy patches on the skin or fatty deposits under the skin, chest pain, shortness of breath, a rapid heartbeat, and white or gray corneal circles. But HoFH, if left untreated, can also lead to heart disease and early death. Some data even suggests that, untreated, HoFH can be fatal as early as age 18. As a result, finding the right treatment option is essential for people living with this condition.
Analyzing the Data to Improve HoFH Care
According to an article in Healio, researchers recently performed a data analysis to predict the impact of adding evinacumab to the current standards-of-care (such as LDL apheresis) for people with HoFH. Their work is published in the European Journal of Preventative Cardiology. The team began by developing a set of mathematical models based on efficacy data from two sources: the Phase 3 ELIPSE HoFH study and peer-reviewed journals for standards-of-care.
Using these models, the team then explored various methods of treatment, such as:
- Monitoring / not pursuing treatment (i.e. untreated individuals)
- High-intensity statins
- Ezetimibe with high-intensity statins
- High-intensity statins alongside ezetimibe and PCSK9i
- Evinacumab with high-intensity statins, ezetimibe, and PCSK9i
Through this evaluation, the research team aimed to understand what the most effective treatment method was, as well as which treatment could best improve outcomes and survival. The mathematical models predicted that, without treatment and depending on other factors such as LDL levels, HoFH was typically fatal between 33 to 43 years old. Using the most robust mathematical model, both high-intensity statin and ezetimibe increased median survival by 9 years, and PCSK9i by 14. When evinacumab was combined with these other treatments, it increased median survival by approximately 12 years.
This builds upon the drug’s benefits as seen in the Phase 3 study, which found that evinacumab reduced LDL levels by nearly half. Given the results of this modeling, the research team suggests that adding evinacumab to treatment plans could fill an unmet need for patients and contribute to longer, healthier lives.
About Evinacumab
Marketed under the brand name EVKEEZA, evinacumab is a monoclonal antibody that is now approved to treat individuals with HoFH who are 5 years old or older. A 2021 news release from Regeneron Pharmaceuticals described the drug as a first-in-class ANGPTL3 inhibitor that was discovered using the company’s proprietary VelocImmune technology. This technology produces fully-human antibodies using genetically engineered mice with humanized immune systems.
Evinacumab may come with side effects such as the common cold, dizziness, arm and leg pain, reduced energy, flu-like symptoms, nausea, and allergic reactions. You can find more information here in the Prescribing Information.
