Inherited or genetic heart disorders, mostly known as Hypertrophic cardiomyopathy (HCM) in medical term, is a condition affecting the left ventricle, the main pumping chamber of the heart. This condition, by which the walls of the left ventricle become thick and stiff, cannot be cured. Over time, the heart can't take in or pump out enough blood during each heartbeat to supply the body's needs.
HCM, usually caused by an inherited genetic variant (familial hypertrophic cardiomyopathy), is passed down through families, can lead to many serious health conditions, such as Atrial fibrillation, a type of arrhythmia that can cause blood clots, heart failure, and stroke.
However, the US drug maker Eli Lilly's new experimental drug, lepodisiran, has shown promising results in reducing levels of lipoprotein(a), the main genetically inherited risk factor for the disease, in a midstage clinical trial.
The drug, when administered in doses of 400 milligrams (either one or two doses), lowered Lp(a) levels by an average of 93.9% over six months, compared to a placebo group. The trial involved 141 patients in the 400 mg group and 69 in the placebo group, with no serious adverse events.
The results of the trial were presented at the American College of Cardiology meeting and published in the New England Journal of Medicine.
"The (Eli Lilly) drug's potential in reducing Lp(a) with infrequent administration, a significant breakthrough since there are currently no approved treatments for elevated Lp(a)," says Dr. Steven Nissen, a cardiologist from the Cleveland Clinic, as quoted by Reuters in one of its related reports on Monday.
HCM is a condition that affects an estimated 1.4 billion people worldwide, including 64 million Americans. Elevated Lp(a) is linked to a higher risk of heart disease, stroke, and other cardiovascular issues, particularly among individuals of African ancestry.
Lepodisiran is one of several drugs being tested to address high Lp(a) levels, and Lilly has already advanced it into late-stage clinical trials. While the drug's ability to lower Lp(a) is a promising development, further large-scale trials are necessary to determine whether this leads to a reduction in actual cardiovascular events like heart attacks or strokes. Lilly is conducting a Phase 3 trial to test this, with patient enrollment expected to be completed this year.