FDA Approves First-Ever Cholesterol-Lowering Pill Targeting PCSK9 for High-Risk Patients

The U.S. Food and Drug Administration (FDA) has approved Lipfendra, a groundbreaking oral medication developed by Merck, marking the first pill designed to lower cholesterol by targeting the PCSK9 protein. Until now, this treatment approach was available only through injectable medicines.

The newly approved drug is intended for patients whose LDL (“bad”) cholesterol remains elevated despite taking statins, the standard treatment for reducing the risk of heart attacks and other cardiovascular diseases.

Lipfendra works by blocking the PCSK9 protein in the liver, allowing the body to remove more LDL cholesterol from the bloodstream. Existing PCSK9 therapies, offered as injections by several pharmaceutical companies, have been effective for years but have faced challenges related to high costs, insurance barriers, and limited patient access.

Merck secured FDA approval after positive results from two clinical trials involving high-risk patients who received Lipfendra in addition to their regular cholesterol-lowering therapy. In one study involving around 3,000 participants, patients experienced a reduction of more than 55% in LDL cholesterol after six months. A second trial showed an average 59% decrease in LDL levels compared with those receiving a placebo.

The cholesterol-lowering effect remained largely consistent over a year of treatment. Researchers reported that side effects, including dizziness and diarrhea, were generally mild and occurred at rates similar to those seen with the placebo. Patients are advised to take the medication on an empty stomach for optimal effectiveness.

The FDA reviewed Lipfendra under its accelerated approval programme for promising therapies that address significant public health needs, enabling a faster regulatory process.

Heart disease continues to be the leading cause of death in the United States, with elevated LDL cholesterol being a major contributor to heart attacks and strokes. While an LDL level below 100 mg/dL is generally considered healthy for most individuals, experts recommend reducing it to 70 mg/dL or lower for people with heart disease or those at high cardiovascular risk.

The approval of Lipfendra offers a convenient oral alternative to injectable PCSK9 inhibitors and could significantly improve access to advanced cholesterol-lowering treatment for patients who require additional LDL reduction beyond statins.

Source: AP