Pricing & dosing controversies: What to expect after Amgen's new PCSK9 approval
- The FDA on Thursday evening approved Amgen's cholesterol-lowering PCSK9 inhibitor Repatha. The drug will be priced at $14,100 per year.
- This is the second FDA approval in this hotly anticipated and likely blockbuster new class of drugs, which drastically reduces patients' LDL cholesterol levels (sometimes by more than 70%). The first was issued to Sanofi/Regeneron's Praluent in July (although Amgen did win the first worldwide approval for a PCSK9 med when the E.U. cleared it last month).
- Repatha is approved for "use in addition to diet and maximally-tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia (HoFH), or clinical atherosclerotic cardiovascular disease, such as heart attacks or strokes, who require additional lowering of LDL cholesterol."
Now that Amgen and Sanofi/Regeneron both have their PCSK9 meds approved, the companies face several major challenges when it comes to pricing and the wisdom of their divergent dosing regimens.
It's clear now that both firms are trying to extract as much money as possible out of a market whose reach may be limited given that the drugs' indications are tailored towards patients whose LDL-C remains uncontrolled despite statin therapy.
The $14,100 (Amgen) and $14,600 (Sanofi/Regeneron) annual price tags have already raised concerns from the two largest pharmacy benefits managers in the country, Express Scripts and CVS Health, and are sure to elicit ardent payer pushback. CVS has said that it will adopt a "wait-and-see" approach for the drugs and that "all bets are off" when it comes to negotiating discounts for them, while Express Scripts has warned that the new therapeutic class could "wreak havoc" on payers.
There are also several critical, unanswered questions regarding dosing and the ultimate efficacy of slashing LDL-C levels by the extent to which Repatha and Praluent do. Amgen is squarely placing its bet on a strategy that involves lowering LDL-C as much as possible, positing that this is the most effective method of mitigating CVD risk. That's why the firm has opted to offer just one dose—the maximum dose attempted in trials.
Sanofi/Regeneron, on the other hand, is offering a bifurcated dosing regimen that includes one high-dose option and one lower-dose option. "This is a new class of drugs," explained Dr. Jay Edelberg, vice president at Sanofi’s PCSK9 development and introduction unit, in an interview with the New York Times. "With any therapy, you always want to treat with the amount of drugs patients need and not more."
It remains to be seen whether doctors are ultimately swayed by the impressive LDL-C lowering numbers associted with Amgen's high-dose drug, or whether or not they want to play it safe and write prescriptions for Praluent since it gives them the option to switch at-risk patients over to a more moderate dose. And only longer-term studies will bear out whether or not this therapeutic class effectively lowers stroke, heart attack, hospitalization, and death risk in patients over the long term. Stay tuned.