New Cholesterol Drug Receives FDA Approval for High Risk Patients

Twitter Summary: @US_FDA approves @Sanofi’s Praluent, first injectable PCSK-9 inhibitor cholesterol-lowering drug

The FDA just approved the drug Praluent, the first cholesterol-lowering treatment in a new class of injectable cholesterol medications called PCSK9 inhibitors. So far, Praluent is approved for two types of patients, in both cases as an add-on for people who can’t take any more statins. These groups are:

• Adult patients with heterozygous familial hypercholesterolemia (HeFH), an inherited condition that causes high levels of LDL cholesterol (aka “bad” cholesterol).

• Adult patients with clinical atherosclerotic cardiovascular disease – a buildup of plaque in the arteries that can lead to conditions such as heart attacks and strokes - who require additional lowering of LDL cholesterol.

Praluent is available in US pharmacies starting today. It’s delivered as a twice-monthly, self-administered 1 mL injection that is available in a 75 mg dose or a 150 mg dose. The cost for either dose is $1,120 for a four-week supply; while this cost may be very expensive for most people, the out-of-pocket cost should be significantly lower for people with insurance plans that cover the drug. The press release from the manufacturers (Sanofi and Regeneron) stated that the companies plan to offer a patient support system that includes copay assistance and free medicine for eligible patients and will help others identify coverage options for out-of-pocket costs.

Clinical trials tested the efficacy and safety of Praluent in patients with HeFH or who were at high risk for heart attack or stroke (~30% of participants in these trials had type 2 diabetes). The participants were randomized to either taking Praluent or a placebo drug (an injection with no active medication), in addition to continuing standard treatments with statins and other lipid-modifying drugs. Participants taking Praluent had a 36%-59% greater average reduction in LDL cholesterol compared to those on the placebo drug, indicating great promise for its use in people with high cholesterol.

Currently, additional clinical trials are examining the effect on the risk of heart disease of adding Praluent to statins. If these trials find the drugs are heart-healthy, the FDA may consider expanding the drug’s approval for a wider population of patients. On that note, while Praluent isn’t designated specifically for people with diabetes, it has the potential to help millions of people with diabetes who have high LDL cholesterol, and we are glad to see the FDA addressing cardiovascular health and supporting innovative options for the treatment and prevention of CVD. -NK/AJW