The FDA has approved alirocumab (Praluent), a drug that gives people who aren’t helped enough by statins a new option to lower their cholesterol and fight heart disease. How many people might that be, however, is a subject of intense scrutiny, as the FDA seems to have given doctors wide discretion in prescribing the drug.
“We do think we have a pretty broad label here,” said Regeneron Pharmaceuticals CEO Leonard Schleifer said on a conference call this afternoon.
The drug was developed by Regeneron (NASDAQ: REGN), of Tarrytown, NY, and French biopharmaceutical firm Sanofi (NYSE: SNY). Regeneron will charge $1,120 for 28 days of the drug in the U.S., which is meant to be injected once every two weeks.That comes out to $560 per dose, or $14,560 a year (assuming 26 doses a year).
The FDA approved two doses, 75mg and 150mg, both to be self-administered by the patient every two weeks. The price will be the same for each, Regeneron said. Insurance companies and their agents—known as pharmacy benefit managers—are expected to push back fiercely and limit the patients they cover. They have warned for months against the costs of alirocumab and similar drugs, which are a new class of cholesterol-cutting agents known as PCSK9 inhibitors.
Officials from CVS Caremark, writing in the journal Health Affairs, warned in February that the PCSK9 drugs could be “the highest selling class of medications in history.”
“Given the number of people potentially eligible for treatment with the PCSK9 inhibitors will number in the millions, the potential overall expenditures by payers are huge,” they wrote, basing their predictions on an annual price of $10,000.
Regeneron’s annual price for alirocumab has come in higher than that. But the company moved quickly today to position itself as sensitive to the potential costs.
On this afternoon’s call, Regeneron’s top commercial executive Bob Terifay said 8 to 10 million people potentially fall under the populations described by FDA—“people who are on statins but not ‘at goal'”—in other words, they’re not at the cholesterol levels their doctors have deemed healthy. But not all those people will be candidates for PCSK9 inhibitors, Terifay said.
Alirocumab is only prescribed for people who have reached a maximum dose on statins but still need lower cholesterol levels. Terifay said Regeneron would have an “education program” to make sure that people taking statins are up to their maximum dose. Such a program could “limit the patient population eligible for PCSK9 inhibitors,” Terifay said.
He also listed other factors that could dissuade alirocumab use—doctors who don’t want to prescribe biologics (PCSK9 inhibitors are monoclonal antibodies), and patients who fear needles, for example.
It also remains to be seen how doctors prescribe alirocumab without “outcomes” data—clinical evidence of better health for patients, not just lower cholesterol. The FDA doesn’t require that evidence for approval. But Sanofi, Regeneron, and developers of rival PCSK9 inhibitors are currently running outcomes trials with tens of thousands of patients that should provide data in a few years.
The FDA has approved alirocumab for people with heterozygous familiar hypercholesterolemia (HeFH), which means they’ve inherited from one parent a genetic condition that causes high levels of LDL, the so-called “bad” cholesterol. One of every 200 to 500 people have this type of FH, according to the FH Foundation, but there is no simple test to determine that genetic status.
The FDA also approved alirocumab for patients already diagnosed with heart disease who need an additional boost beyond statins to lower their cholesterol. The agency’s specific language—“patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol”—is what has triggered initial estimates of a broad potential population.
“We believe that the label is broad, beyond HeFH patients only, and likely to have a few to several million patients as an eligible market,” RBC Capital Markets analyst Adnan Butt wrote in a research note.
Adding to the speculation, the agency doesn’t specifically address people who are intolerant to statins. Those folks represent anywhere from five to 20 percent of the high-cholesterol population.
Ethan Weiss, a cardiologist at the University of California, San Francisco, says the patient population described by the FDA would include “almost all” of his patients, leaving “lots of room for nuance (and) judgment” when prescribing the drug. “Payers will be the gatekeeper,” Weiss says, and he predicts they will make the decision to prescribe alirocumab (and other PCSK9 inhibitors, if approved) “as painful as possible.”
The agency is also considering evolocumab (Repatha), from Amgen (NASDAQ: AMGN), which also lowers cholesterol by blocking PCSK9. The deadline for the FDA decision on evolocumab is August 27.
European regulators approved evolocumab earlier this week and are likely to approve alirocumab, as well. The competition is so fierce that Regeneron and Sanofi paid $67.5 million in 2014 for a voucher that let them speed up the FDA review of alirocumab by a few months, which is why their approval date came up one month before Amgen.
Attention now turns to CVS Caremark, Express Scripts, and other drug-buying groups to see how they react to the label and the pricing.
Image courtesy of the National Library of Medicine.