On the brink of FDA approval is a new class of cholesterol-lowering drugs that have the potential to change the landscape in the prevention of heart attack and stroke.
"These new drugs are a blockbuster as far as achieving the goal of getting LDL levels down markedly," said Thomas Whayne, MD, PhD. "Particularly in the cases where high-risk patients cannot tolerate the current standard of care, we will have a powerful pharmaceutical alternative. It's going to be fantastic."
Called PCSK9 inhibitors, these drugs appear to sharply reduce levels of bad (LDL) cholesterol. Clinical trials of PCSK9 inhibitor drugs developed by Amgen, Sanofi and Regeneron demonstrate LDL levels are reduced by 60%. By comparison, statins -- the current class of drugs that are the mainstay of treatment for high LDL -- generally reduce LDL levels by 30-50%.
High LDL levels are associated with increased risk for heart disease and stroke. Given that nearly a third of all U.S. adults have high LDL, the market for these drugs is expansive.
Many in the medical field caution that there's no solid long-term evidence that these robust reductions in LDL levels actually translate to fewer strokes, heart attacks or other CV events, but Dr. Whayne is optimistic.
"There is data going back 40+ years demonstrating without reservation that reducing LDL levels is of benefit in lowering cardiovascular risk," said Dr. Whayne. "While it's always prudent to have long-term safety and efficacy data, I think bringing this drug to market now makes good sense."
A Food & Drug Administration advisory committee reviewed the clinical trial data this week and recommended that the FDA approve PCSK9 drugs by a 13-3 vote. A decision from the FDA whether to allow the drugs to go on the market should come by the end of July, but the agency usually follows the advice of its committees.
One drawback to the PCSK9 inhibitors is the $10,000 per year price tag. Dr. Whayne is concerned that insurers will be hesitant to approve PCSK9 inhibitors to patients when statins are far less expensive.
"Statins will still have their place in the medicine cabinet," said Dr. Whayne, "but having a whole group of other approaches for the patient who simply can't tolerate statins but is at extremely high risk...this could be very important. I can see myself doing battle with the pharmacy benefits companies to get this drug approved for those patients."
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