A Food and Drug Administration advisory committee Wednesday recommended that the agency approve the cholesterol-lowering drug Repatha from Amgen Inc., the second in a class of promising heart medicines that could change medical practice and generate billions of dollars in sales.

The panel voted 11-4 to recommend the FDA approve Repatha, saying its benefits outweighed its risks. But panel members expressed concerns that Amgen primarily relied on studies that lasted only 12 weeks.

The committee also voted 15-0 to recommend approval of Repatha's use in a rare subset of familial hypercholesterolemia, a genetic disease causing extremely high cholesterol. The population of this subset, called homozygous familial hypercholesterolemia, has been estimated as low as one in 1 million people.

The overall approval recommendation would endorse injections either monthly or every two weeks.

The panel voted on the injectable Amgen drug a day after it recommended approval of the drug Praluent, from Sanofi SA and Regeneron Pharmaceuticals Inc. The committee members expressed concerns both days about the fact that both drugs' FDA applications are largely based on lowering LDL, or "bad" cholesterol, not on avoiding bad outcomes like heart attacks. The panelists worried that, while LDL cholesterol has proven a good early measure—or "surrogate endpoint"—of whether statin drugs like Lipitor reduce heart disease, that may not be the case with this new class of medicines.

"It would be important to have clinical evidence of benefit, and a surrogate is not adequate," said panel member William R. Hiatt, a cardiologist at the University of Colorado. Some other panelists felt that patients with extremely high cholesterol need a powerful, cholesterol-lowering agent.

Amgen stock rose 0.5% in after-hours trading to $156.25.

Repatha, generically called evolocumab, "results in statistically significant reductions in LDL cholesterol of approximately 60% after 12 and 52 weeks of treatment," an FDA analysis of clinical-trial data said.

Reviewers from the FDA noted that historically, "reduction of LDL cholesterol alone has been viewed favorably as a surrogate outcome if the reduction was sufficiently robust" and there weren't safety red flags linked to the drug. But, the analysis said, there have been some trials in recent years showing that lowering LDL doesn't always reduce heart disease.

Some prominent cardiologists who weren't on the committee said the way the Amgen drug works is similar enough to that of statins, so that lowered LDL cholesterol should mean better outcomes.

Steven E. Nissen, chairman of cardiovascular medicine at the Cleveland Clinic, said, "There are some times that surrogate endpoints are useful." This is such a case, he said in an interview.

One panel member, Philip Sager of Stanford University, said he voted in favor of approving the drug "because of the large, unmet medical need."

But Martha Nason of the National Institute of Allergy and Infectious Diseases voted against recommending approval. "I don't put much stock in 12-week safety data," she said.

The FDA often follows the recommendations of its advisory panels, but it isn't required to do so.

Amgen has already fully enrolled a study of 27,500 patients to see if Repatha does in fact lower the rate of heart attacks and other cardiovascular events compared to statin drugs alone. Amgen said the study should produce results by 2017.

Several speakers at the FDA advisory hearing were patients with the genetic high-cholesterol disorder, who said they need more treatment options.

Regarding safety, an FDA reviewer said "there were no marked disparities in deaths, serious adverse events or adverse events leading to discontinuation" of the Amgen drug. However, FDA officials speaking at the panel meeting expressed some reservations about the relatively short, 12-week duration of the completed, controlled Repatha studies, involving 3,152 patients.

The 60% reduction in LDL within weeks is unusual for a drug and also very hard for patients to achieve with diet and exercise alone. Still, the question is whether that reduction, from a drug working in a novel way, reduces disease. The PCSK9 drugs work by blocking the protein called PCSK9 that interferes with the liver's ability to clear bad cholesterol from the bloodstream.

"Regardless of how confident we may be in the 'LDL hypothesis,' we must remember that LDL cholesterol remains a surrogate and not a clinical outcome that reflects how patients feel, function or survive," wrote James P. Smith, a doctor and deputy director of the FDA drug center's division of metabolism and endocrinology products.

Write to Thomas M. Burton at tom.burton@wsj.com

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