U.S. regulators won’t require Amgen Inc., Sanofi, Pfizer Inc. and other companies developing the next class of cardiovascular medicines to prove the therapies prevent heart attacks and death before they’re approved.
The drugs, known as PCSK9 inhibitors, will only have to meet the U.S. Food and Drug Administration’s existing standards for clearance, including whether they cut cholesterol and reduce blood pressure or inflammation, said Eric Colman, a deputy director in the drug evaluation and research branch.
The issue was raised after cholesterol care guidelines released this week said people should only take statins to reduce their levels, since other drugs hadn’t specifically been proven to reduce cardiac complications. While the new drugs, which work differently from statins, cut cholesterol in studies, they haven’t completed broader trials that would prove they help people avoid heart attacks or death.
“There is a race to the finish line to get these out the door,” said Ira Loss, an analyst at Washington Analysis that tracks the FDA.
Analysts have said the PCSK9 inhibitors, which block a protein that prevents liver cells from processing LDL and clearing it from the body, will generate $2 billion in 2018. Broad-scale trials to prove medical outcomes can involve more than 20,000 patients, cost as much as $500 million and add years on to the development process.
Shares of PCSK9 developers rose. Tarrytown, New York-based Regeneron Pharmaceuticals Inc., which is developing its medicine with French drugmaker Sanofi, jumped 4.2 percent to $288.83 at the close in New York. Amgen, based in Thousand Oaks, California, gained 1.7 percent to $115.41. Pfizer, based in New York, rose less than 1 percent to $32.
An FDA approval decision “will be based on the compound’s effects on the entire lipoprotein lipid panel, particularly LDL-cholesterol, its effects on other markers of potential cardiovascular risk,” such as blood pressure, as well as any problems with toxicity, Colman said in an interview.
The new cholesterol guidelines from the American College of Cardiology and the American Heart Association were released Nov. 12. They recommend only statin drugs such as Pfizer’s Lipitor and AstraZeneca Plc’s Crestor since studies involving more than 190,000 patients conclusively prove their benefit. Rivals that work differently, including Merck’s Zetia and Vytorin, haven’t been shown to reduce the risk of heart complications and aren’t backed for routine use.
The PCSK9 inhibitors are monoclonal antibodies, proteins grown in living cells that are injected into the body to target an enzyme produced by the PCSK9 gene. They were developed to help people who can’t tolerate statins, or who don’t get their cholesterol under control with older medicines, a group that encompasses about 12 million people in the U.S.
Roche Holding AG, Merck & Co., Alnylam Pharmaceuticals Inc. and Eli Lilly & Co. all have PCSK9 inhibitors in their product pipelines.
Heart disease remains the leading cause of death worldwide, killing more than 600,000 Americans every year, according to the U.S. Centers for Disease Control and Prevention. High levels of bad LDL cholesterol, which can form into fatty plaque, raises the risk that the arteries will clog and cause a heart attack.
For now, the FDA says it’s sticking with the traditional approach to approving cholesterol medications. The results of a trial with Merck & Co.’s Vytorin, called Improve-It, has the potential to change the equation if the drug’s cholesterol lowering ability fails to translate into longer life and fewer heart attacks, said Colman, the FDA’s deputy director of the division of metabolism and endocrinology products.
“Looking ahead, the results of the ongoing trial Improve-It will provide important insight into the incremental effect on risk for major adverse cardiovascular events of adding a non-statin, LDL-cholesterol lowering drug to subjects receiving background statin therapy,” Colman said.