FDA Approves Praluent for Treatment of High Cholesterol

Annals of Long-Term Care: Clinical Care and Aging. 2015;23(8):47

The FDA recently approved a new injection-based treatment for high cholesterol that was developed by Sanofi and Regeneron Pharmaceuticals (https://1.usa.gov/1CZvYDg). The drug, named Praluent, is recommended for adult patients in conjunction with a healthy diet and maximally tolerated statin therapy. Praluent will be the newest addition to a number of different drugs and treatment approaches for hypercholesterolemia (HeFH), which is linked to cardiovascular disease, the number one cause of death for Americans.

Praluent is an antibody that targets the specific protein PCSK9 by blocking its access to certain receptors in the liver. More receptors are then available to clear low-density lipoprotein (LDL) cholesterol from the blood to prevent negative outcomes like cardiovascular disease. In five placebo-controlled trials, a total of 2476 participants—all of whom had either HeFH or high risk for heart attack or stroke—were treated with Praluent. Participants using the injection had average reductions in LDL cholesterol of 36–59%, greater than in those using placebo.

Currently, statins are the drug most often used to curb high cholesterol levels; and, whereas Praluent is a biweekly injection that will cost users about $14,600 a year, statin therapy can cost patients only a few cents per pill. However, reports on statin therapy indicate only a 25–55% percent reduction in cholesterol levels, and some patients have complained of fatigue and muscle aches when taking the drug.¹ Trials evaluating the addition of Praluent to statin therapy for the reduction of cardiovascular risk are still ongoing. Additionally, in patients with severe cholesterol levels, such as those diagnosed with familial hypercholesterolemia, statin therapy may be marginally ineffective, forcing them to undergo apheresis, a grueling, $8000 per month treatment that requires the patient to sit for hours as his or her blood is pumped through a machine and back into his or her body.1

Dr. James Underberg, MD, professor at the NYU Center for Cardiovascular Disease Prevention (New York, NY) and president-elect of the northeast chapter of the National Lipid Association, considers Praluent’s approval to be a significant step in the right direction for the treatment of high cholesterol, calling Praluent one of the “first new compounds available for the management of patients with elevated cholesterol in almost a decade.”

“[PCSK9 inhibitors] lower LDL and total cholesterol as much as, and in some cases more than, potent statins, and seem to have minimal safety issues... [They] offer a reasonable option for additional cholesterol lowering for those patients with inherited cholesterol disorders (Familial Hypercholesterolemia) and/or symptomatic atherosclerotic cardiovascular disease (ASCVD) who are unable to attain the desired cholesterol goal on optimized statin therapy,” Dr. Underberg said by email. He added that, although Praluent and those like it may be more expensive than other drugs on the market, for the patients who need it, Praluent “represent[s] a new option in care.”

The most common side effects of Praluent include itching, swelling, pain, or bruising around the injection sight, nasopharyngitis, and flu. There have also been some reports of hypersensitivity vasculitis (a skin rash usually appearing as purple-colored spots on the skin) and hypersensitivity requiring hospitalization. Patients who experience serious allergic reactions should immediately stop using Praluent and seek medical attention. —Sean McGuire

References:

  
1.    Kolata G. Praluent Looks Cheap to Those With Extreme Cholesterol. New York Times. July 27, 2015. https://nyti.ms/1gj6vLd. Accessed July 31, 2015.