A Food and Drug Administration advisory panel has recommended the approval of a new injectable drug that could help prevent heart attacks and other cardiovascular health problems by greatly lowering cholesterol. The panel voted 13-3 in favor of Praluent — one of a new wave of medicines that target LDL cholesterol — but scientists note that definitive evidence that the drug can reduce heart problems will have to wait until full clinical trials are completed in 2017.
The drug appears to cause no major side effects
Praluent works by blocking a protein called PCSK9 that normally stops the liver from being able to fully clear LDL cholesterol from the bloodstream. Tests have shown that by taking the new drug, patients have reduced their cholesterol by between 40 and 65 percent, even if they have previously been taking cholesterol-managing statins such as Lipitor. The drug also appears so far to have no significant side effects on patients who do take it — the FDA has questioned whether it could cause forgetfulness or delirium, but appears to be satisfied with the safety studies conducted so far.
Speaking to The New York Times, Harvard cardiologist Peter Libby said that drugs such as Praluent mean that "the LDL issue is solved in principle." But due to a lack of data, others in the medical field have urged caution in the use of the new medicine. So-called "bad" cholesterol has been shown to be significant in cardiovascular scientific studies, but the direct links between lowering LDL cholesterol and reducing the risk of heart disease are still not concrete. Brendan M. Everett, cardiologist at Boston's Brigham and Women's Hospital, told The Wall Street Journal he would only use the drug for patients genetically predisposed to high levels of cholesterol and avoid broader use.
Some people with genetic mutations that also block the PCSK9 protein live perfectly healthy lives with very low cholesterol. But what is the effect on the body when someone with previously dangerous levels drops down to such a low LDL level? Should Praluent and its ilk be prescribed to anyone with heightened cholesterol, or saved only for people with critical levels in their bloodstream? And when statins have also been successful in reducing cholesterol to some degree, how much will this new, supposedly more effective treatment cost?
A number of new cholesterol-reducing drugs are aiming to enter the market
The product is one of two major new cholesterol-reducing drugs set to enter the market. Praluent — generic name alirocumab — was developed by Sanofi and Regeneron Pharmaceuticals, while another similar drug, called Repatha, is the work of medical firm Amgen. Both companies estimate that their drugs will help millions in the US, but both have kept quiet about the price of a year-long course of their medications. Dr. William Shrank, chief scientific officer at CVS Health, estimates the cost to the patient at between $7,000 and $12,000 a year. If the drugs are only given to people who can't reduce their cholesterol low enough with statins such as Lipitor, Shrank told The New York Times, then the cost would be $16 billion. If it was widened out to encompass everyone with a history of heart disease, that price rises to $186 billion.
Amgen's Repatha goes up against the FDA's advisory board today. While the administration doesn't technically have to follow the board's advice, it historically has, indicating that Praluent at least is likely to be approved. The government body does say, however, that if the large-scale trials underway by both companies don't show proof that the drug can dramatically reduce cholesterol and in turn prevent heart disease, then it will rescind that approval in a few years.