The United States Food and Drug Administration (FDA) has approved the first oral PCSK9 inhibitor, offering a new option for patients with high bad cholesterol. Developed by Merck pharmaceutical company, the tablets will be sold under the brand name Lipfendra. The pill is intended for people whose cholesterol levels remain high despite taking statins, the standard treatment for reducing heart attack risk.
Unlike existing PCSK9 therapies, which require injections, Lipfendra comes in tablet form. The medicine works by blocking the liver protein PCSK9, which limits the body’s ability to remove low-density lipoprotein (LDL), commonly known as bad cholesterol.
Consequently, the approval provides a more convenient treatment option for eligible patients.
Clinical trials show significant cholesterol reduction
Merck received FDA approval after completing two clinical studies involving patients at high risk of cardiovascular disease. Participants continued taking statins while adding Lipfendra to their treatment plans.
In one trial involving 3,000 patients, the pill reduced LDL cholesterol levels by more than 55 percent after six months. Meanwhile, a second study found an average reduction of 59 percent compared with patients who received a placebo. Researchers also reported that the treatment’s benefits remained largely stable over one year.
Furthermore, side effects, including dizziness and diarrhoea, occurred at similar rates in both the treatment and placebo groups. However, patients must take the pill on an empty stomach for the best results. Existing PCSK9 medicines have remained difficult to access because of high costs, insurance restrictions, and limited prescribing.
Experts believe the first oral PCSK9 inhibitor could improve access to advanced cholesterol-lowering treatment for millions of patients in the world.
