New drugs in development are offering hope for people with high levels of lipoprotein(a), or Lp(a), a hereditary cholesterol-related condition that increases the risk of heart attack or stroke. This condition, affecting 1 in 5 people, often goes undetected until a cardiovascular event occurs. Testing for Lp(a) is not yet routine but has been gaining traction, with experts recommending everyone be tested at least once in their lifetime.
Several drugs targeting Lp(a) are in clinical trials, with promising results. Among them, Pelacarsen, an injectable medication administered monthly, reduced Lp(a) levels by up to 80% in earlier trials. The study is expected to conclude in May 2025. Another injectable, Olpasiran, showed a 95% reduction in Lp(a) levels in phase II trials and is administered quarterly. Its final results are anticipated in 2026. Lepodisiran, still in early trials, demonstrated a sustained 94% reduction with a single injection. This drug is unique as it includes participants who have not yet experienced a cardiovascular event. However, results are not expected until 2029.
Muvalaplin, a daily pill, offers a different approach by interfering with the assembly of Lp(a) outside the cell. In phase II trials, it reduced Lp(a) levels by 86%. Zerlasiran, another injectable, showed similar reductions in phase II trials, but phase III details remain pending.
Unlike other medications, Muvalaplin’s pill form may make it more accessible and cost-effective. It also avoids lingering in the liver, which could reduce the risk of long-term side effects. This makes it an attractive option for individuals with liver conditions.
The ultimate goal of these trials is to confirm whether lowering Lp(a) levels reduces the risk of heart attacks and strokes. While researchers are optimistic, they emphasize the importance of large-scale studies to confirm these benefits. Previous experiences with cholesterol treatments have shown that reducing levels does not always translate to fewer cardiovascular events. However, genetic evidence strongly supports the connection between Lp(a) and cardiovascular risk.
For individuals with high Lp(a), the psychological burden of the condition can be significant. Many adopt strict lifestyles to manage their health, including a heart-healthy diet, regular exercise, and multiple medications. The prospect of effective treatments offers hope for a better quality of life and reduced anxiety about potential health crises.
Despite the potential of these treatments, awareness of Lp(a) remains low. Testing is not part of routine lipid panels, and many people learn about their condition only after a cardiovascular event. Advocates encourage everyone to request this simple blood test to assess their risk.
For those affected, these developments represent more than medical progress—they symbolize hope for a future where managing Lp(a) is no longer a daunting challenge. Families affected by hereditary cholesterol disorders look forward to seeing these treatments bring meaningful change to their lives.
