By Kate Greenwood
[Cross-posted at Health Reform Watch]
This summer, the Food and Drug Administration (FDA) is expected to approve the first entries in a new class of drugs that lower patients’ low-density lipoprotein (LDL) cholesterol levels by more than half, even those patients who are already taking other cholesterol-lowering medication. The new drugs are biologics—monoclonal antibodies—that target, and inhibit, the gene proprotein convertase subtilisin–kexin type 9 (PCSK9). In mid-March, the New England Journal of Medicine published the results of important studies (here and here) of the two PCSK9 inhibitors that the FDA is expected to pass judgment on this summer. These studies—of Repatha (evolocumab), which is sponsored by Amgen, and Praluent (alirocumab), which is sponsored by Sanofi and Regeneron—suggest, but do not definitively establish, that PCSK9 inhibitors will reduce not just LDL levels, but also a patient’s chance of a major cardiovascular “event”, like a heart attack or stroke.
The race to approval between Amgen and Sanofi and Regeneron has been dramatic. (Pfizer also has a monoclonal antibody PCSK9 inhibitor in development, but it has lagged behind the two leaders.) As John Carroll reported at FierceBiotech last summer, Sanofi and Regeneron jumped ahead of Amgen when they purchased a priority review voucher from BioMarin for $67.5 million dollars. BioMarin was awarded the priority review voucher, which shrinks the time the FDA takes to approve a drug from ten months to six, because it developed and sought approval for a treatment for a rare pediatric disease. Per the Wall Street Journal, “[t]he voucher was the first to be issued under the pediatric incentive program, and also the first to change hands.”
As I mentioned earlier this week here, speculation has begun about what the price of the new PCSK9 inhibitors will be. Weighing in favor of a high price, the evidence of their efficacy is impressive and growing. The drugs hold particular promise for patients who cannot tolerate statin medications, or whose cholesterol cannot be controlled by statins alone. And, they are biologics, which are more expensive to produce than small-molecule drugs. On the other hand, PCSK9 inhibitors are not without safety concerns. In addition, patients will have to inject themselves with the new drugs, which some will find undesirable (although some might prefer a once- or twice-a-month injection to a daily pill regimen). Finally, the new drugs will have to compete with generic statins.
Payers are very concerned. Continue reading