As Esperion Therapeutics steered its cholesterol-lowering drug through clinical testing and regulatory review, CEO Tim Mayleben did something unusual for a pharmaceutical executive: he spoke openly about price. Specifically, he talked about how inexpensive the drug would be.
As early as two years ago, Mayleben said an FDA-approved Esperion (NASDAQ: ESPR) drug would cost patients about $10 per daily pill—a fraction of the price of newer cholesterol drugs that have reached the market in recent years. The FDA approved the Esperion drug, bempedoic acid (Nexletol), late Friday. On Monday, Mayleben said the drug will cost patients even less than he had projected. A patient’s $10 out-of-pocket cost will cover a three-month supply.
“We are totally focused on patient affordability and access to our medicine,” Mayleben said on a conference call to discuss the drug’s approval.
The FDA approved bempedoic acid for adults who have heterozygous familial hypercholesterolemia, an inherited disorder that leads to high levels of LDL cholesterol, or the “bad cholesterol.” The approval also covers patients who have atherosclerotic cardiovascular disease, a buildup of cholesterol plaque in the arteries that requires additional therapy to lower cholesterol levels.
The Esperion drug is meant to supplement diet and exercise, as well as statin therapy, an older class of cholesterol-lowering drugs. Mayleben noted that not all patients can tolerate statins and for some of those who can, these drugs don’t work well enough. Those patients, representing about 15 million people in the US, are the main target for Esperion’s therapy, he said.
Bempedoic acid is a small molecule drug intended to block cholesterol synthesis in the liver, similar to the approach of statins. But statins are also thought to affect cholesterol synthesis in skeletal muscle. Muscle pain and weakness are among statin’s side effects, and these problems can lead patients to stop taking the drugs. That’s one of the reasons companies have been trying to develop pharmaceutical alternatives.
The FDA approval of bempedoic acid is based on two Phase 3 tests that enrolled more than 3,000 patients. Esperion reported that patients treated with its drug showed an average LDL-cholesterol reduction of 18 percent on top of statin therapy. But bempedoic acid also comes with some risks. But the drug’s label warns that patients treated with the drug may develop hyperuricemia, or high levels of uric acid in the blood. Other problems reported include tendon rupture.
Besides high uric acid levels, the most common side effects reported in the studies included upper respiratory tract infections, muscle spasms, back pain, abdominal pain, bronchitis, and elevated liver enzymes. Esperion says that most of the side effects were mild to moderate, and in line with what was reported in the placebo group. The label also cautions that the effect of bempedoic acid on the incidence of cardiovascular problems or even death has not been determined. A cardiovascular outcomes clinical trial is currently underway.
Approval of bempedoic acid puts that drug on track to compete against another new class of cholesterol-lowering medicines, antibody drugs known as PCSK9 inhibitors. Unlike Esperion’s drug, PCSK9 inhibitors are given as injections. Amgen (NASDAQ: AMGN), and partners Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi (NYSE: SNY), have a nearly five-year head start with their FDA-approved PCSk9 drugs. But after launching with $14,000 a year price tags, sales of those drugs were slow to take off. That led the companies to slash their prices.
Meanwhile, (NYSE: NVS) joined the cholesterol drug fray last fall with its $9.7 billion acquisition of The Medicines Company, whose drug inclisiran stops PCSK9 by silencing the gene that produces the protein. That drug is also injectable, but developed to be given twice a year versus monthly for the currently approved PCSK9 inhibitors. Similar to the comments made by Esperion’s Mayleben, executives for The Medicines Company had spoken publicly about how inclisiran could be less expensive than competing drugs.
Mayleben said Esperion plans to launch its drug on March 30. In addition to having a more convenient tablet formulation compared to the injectable PCSK9 drugs, Esperion’s drug will have a pricing advantage. The $10 price for a three-month supply is for patients with insurance. For others, the company will offer co-pay and payment assistance programs to bring a patient’s three-month supply costs to $10. Mark Glickman, Esperion’s chief commercial officer, said on the conference call that the company expects to keep pricing at that level in order to ensure patient access to the drug.
Esperion is also awaiting a regulatory decision for another drug. The company had tested a pill that combines bempedoic acid with ezetimibe, a generic cholesterol-lowering medication. An FDA decision for that combination treatment is expected on Wednesday.
Image: iStock/Rasi Bhadramani