What Is Evinacumab and How Can It Help People With HoFH?
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Homozygous familial hypercholesterolemia (HoFH) is a rare and serious inherited condition where people are born with very high levels of bad cholesterol (LDL-C). This makes them more likely to have heart problems at a young age. Regular cholesterol-lowering medicines like statins often don’t work well enough for them. That’s where a new drug called evinacumab comes in.
What Is Evinacumab?
Evinacumab is a special type of medicine called a monoclonal antibody. It targets a protein called ANGPTL3, which normally increases cholesterol levels in the blood. By blocking this protein, evinacumab helps lower cholesterol in people with HoFH. It is given through a monthly intravenous (IV) infusion and is approved for children as young as 5 years old.
Why Is This Drug Important?
People with HoFH struggle to lower their cholesterol even when they take several medications. High LDL-C levels put them at serious risk of heart attacks and other heart problems. Evinacumab offers an extra layer of help by lowering LDL-C by nearly 50 percent, even in people already on multiple cholesterol-lowering treatments.
What Did the Real-World Study Show?
A new study looked at how evinacumab works outside of clinical trials, in real-life situations. Researchers followed 24 patients with HoFH, both children and adults, from six medical centers in the US. These patients were already taking other treatments for cholesterol when they started using evinacumab.
Here’s what they found:
- LDL-C dropped by about 53 percent after using evinacumab for nearly a year
- Non-HDL cholesterol, total cholesterol, and triglycerides also dropped significantly
- About 30 percent of patients reached the target LDL-C level of under 70 mg/dL
- The results were seen as early as four weeks after starting treatment
Were There Any Side Effects?
Yes, but most of them were mild. About 38 percent of patients had side effects like dizziness, nausea, or flu-like symptoms during or after their infusion. Only one person stopped the treatment due to back pain. In some cases, doctors used antihistamines or slowed down the infusion to help patients feel better.
Is It Safe for Children?
Yes, evinacumab is now approved for children as young as 5. In smaller studies involving children, it showed similar benefits to adults in lowering LDL-C. While some children experienced side effects like sore throat or stomach upset, most continued with treatment.
How Does It Compare to Older Studies?
The results from this real-world study match what was seen in earlier clinical trials. In one large trial, evinacumab lowered LDL-C by nearly 50 percent. Side effects were also similar, and most were mild.
What’s the Bottom Line?
Evinacumab is an important option for people with HoFH who are not getting enough help from other cholesterol medicines. It works well in both children and adults, and it is generally safe. While some people may have mild side effects, the benefits in reducing cholesterol and protecting the heart are clear.
What Should Patients and Families Know?
- Evinacumab is not a first-line treatment. It is added when standard treatments are not enough
- It requires regular monthly IV infusions
- Insurance coverage may vary and can be a challenge
- Patients should work closely with a specialist to monitor their progress and manage any side effects
Final Thoughts
For people living with HoFH, evinacumab offers hope. It helps lower dangerously high cholesterol levels when other treatments fall short. With more real-world data now available, doctors can better understand how to use this medication safely and effectively for patients who need it most.