The FDA approved the new cholesterol-lowering drug Repatha (evolocumab) on Thursday.
The injectable drug from Amgen is the second in a new class of drugs called PCSK9 inhibitors. It works by making the liver more efficient at getting rid of LDL, or bad cholesterol. High levels of LDL cholesterol in the blood are linked to heart disease.
Repatha is approved for patients who have heterozygous or homozygous familial hypercholesterolemia, HeFh or HoFh, which is an inherited condition that causes high LDL cholesterol levels. It is also approved for patients who have had a heart attack or stroke.
The drug, which is given as an injection either once every two weeks or once a month in a higher dosage can be self-administered through a prefilled auto-injector pen or syringe.
This offers another treatment for patients who aren’t responding to currently available medications or who can’t take them because they experience side effects.
“Repatha provides another treatment option in this new class of drugs for patients with familial hypercholesterolemia or with known cardiovascular disease who have not been able to lower their LDL cholesterol enough with statins,” Dr. John Jenkins, director of the FDA Office of New Drugs, Center for Drug Evaluation and Research, said in a news release.
The currently available medications for lowering cholesterol are statins, of which there are seven on the market. Patients who aren’t responding to statins alone would continue taking them in addition to Repatha and maintaining a healthy, cholesterol-friendly diet. Patients who can’t tolerate statins because of the side effects would take just Repatha.
Dr. Sean Harper, Amgen’s executive vice president of research and development, said it makes sense that statins are the first line of treatment, but millions of patients still have high LDL that needs to be lowered.
“These are patients who are ticking time bombs. … Their doctor is waiting to see what their next (cardiac) event is. Will it be heart attack? Stroke? Chest pain in the emergency room?”
The drug was recommended by an FDA advisory committee in June and was approved in Europe last month.
In July, Sanofi and Regeneron’s Praluent (alirocumab) became the first PCSK9 inhibitor approved in the United States.
Repatha will be available next week, according to Amgen, at a wholesale cost of $542.31 for a single dose to be given once every two weeks. That’s $14,100 a year. The cost for individual patients will vary depending on insurance coverage.
In comparison, statins cost between $500 and $700 a year for name-brand versions and $48 a year for generics.
The most common side effects are inflammation in the nasal or throat passages, upper respiratory tract infections, flu and black pain. Patients may also experience injection site reactions including redness, pain and bruising. Hives have also been reported by patients who have taken Repatha.
Thursday was the deadline for the agency to make a decision on whether to approve the drug.
These new drugs are “a powerful new way of lowering the bad form of cholesterol, and that has profound implications in dealing with the burden of vascular disease,” which can lead to heart attacks and stroke, Dr. Elliott Antman, president of the American Heart Association, said in June.
Antman and others have speculated that while these drugs are limited to patients who can’t take statins or aren’t responding to them, down the road they could be more broadly prescribed for people with high cholesterol.
Not to be outdone, Pfizer is preparing to enter this market, too, with their own PCSK9 called Bococizumab. They have designed eight clinical trials, some of them currently underway. Unlike the two drugs now approved, which are injections, Pfizer is researching an injection, a pill and a vaccine that would prevent high cholesterol using immunotherapy.
