FDA Approves Repatha

• FDA approved: Yes (First approved August 27th, 2015)
• Brand name: Repatha
• Generic name: evolocumab
• Company: Amgen Inc.
• Treatment for: High Cholesterol, Familial Heterozygous, High Cholesterol, Familial Homozygous,High Cholesterol

Repatha (evolocumab) is a monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) for the treatment of patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, or patients with atherosclerotic heart disease who require additional lowering of LDL-cholesterol.The FDA approved Amgen’s Repatha (evolocumab) for U.S. marketing on August 27, 2015. Repatha injection is indicated for use in addition to diet and maximally-tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia (HoFH), or clinical atherosclerotic cardiovascular disease (ASCVD), such as heart attacks or strokes, who require additional lowering of LDL cholesterol.

Prior to full approval, Repatha underwent FDA advisory committee review on Wednesday, June 10th, 2015. The FDA committee voted 11-4 in favor of FDA approval, including a 15-0 approval for homozygous familial hypercholesterolemia (HoFH).In Phase 3 trials evolocumab lowered LDL-C by roughly 60% across all study groups in 12- and 52-week studies; in HoFH, evolocumab reduced LDL-C by 31%. FDA briefing documents lists further clinical study details.

Common side effects with Repatha use include nasopharyngitis (common old), upper respiratory tract infections, influenza, back pain, and injection site reactions like redness, pain or bruising. Allergic reactions, such as rash and hives, have been reported. Patients should stop using Repatha and seek emergency medical care if they experience symptoms of a serious allergic reaction, such as swelling of the lips, tongue or throat.Repatha is the second PCSK9 inhibitor drug to be approved this summer. Sanofi-Aventis and Regeneron’s Praluent (alirocumab) was given the agency go-ahead on July 24, 2015 for treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein (LDL) cholesterol.

The recommended dose of Repatha for adults is 140 mg every two weeks or 420 mg once a month. Repatha is available as a single-use 140 mg prefilled autoinjector or prefilled syringe that patients can self-administer. For adults with HoFH, the recommended dose is 420 mg once a month.Repatha is expected to be available in the U.S. next week. According to Amgen, the Wholesale Acquisition Cost (WAC) of Repatha is $14,100 annually for the every two weeks administration. Actual costs to patients, payers and health systems are anticipated to be different, based on pricing negotiations, insurance coverage or patient assistance programs.Repatha, the second drug approved in a new class of drugs known as PCSK9 inhibitors, is approved for use in addition to diet and maximally-tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia (HoFH), or clinical atherosclerotic cardiovascular disease, such as heart attacks or strokes, who require additional lowering of LDL cholesterol.

Familial hypercholesterolemia (encompassing both HeFH and HoFH) is an inherited condition that causes high levels of LDL cholesterol. A high level of LDL cholesterol in the blood is linked to cardiovascular or heart disease. Heart disease is the number one cause of death for Americans, both men and women. According to the Centers for Disease Control and Prevention, about 610,000 people die of heart disease in the United States every year– that equals one in every four deaths.

“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,” said John Jenkins, M.D., director of the Office of New Drugs, Center for Drug Evaluation and Research. “Cardiovascular disease is a serious threat to the health of Americans, and the FDA is committed to facilitating the development and approval of effective and safe drugs to address this important public health problem.”Repatha is an antibody that targets a specific protein, called PCSK9. PCSK9 reduces the number of receptors on the liver that remove LDL cholesterol from the blood. By blocking PCSK9’s ability to work, more receptors are available to get rid of LDL cholesterol from the blood and, as a result, lower LDL cholesterol levels.

The efficacy and safety of Repatha were evaluated in one 52-week placebo-controlled trial and eight 12-week placebo-controlled trials in participants with primary hyperlipidemia, including two that specifically enrolled participants with HeFH and one that enrolled participants with HoFH. In one of the 12-week studies, 329 participants with HeFH, who required additional lowering of LDL cholesterol despite statins with or without other lipid-lowering therapies, were randomized to receive Repatha or placebo for 12 weeks. Participants taking Repatha had an average reduction in LDL cholesterol of approximately 60 percent, compared to placebo.

The most common side effects of Repatha include nasopharyngitis, upper respiratory tract infection, flu, back pain, and reactions such as redness, pain, or bruising where the injection is given. Allergic reactions, such as rash and hives, have been reported with the use of Repatha. Patients should stop using Repatha and get medical help if they experience symptoms of a serious allergic reaction.Multiple clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke. A trial evaluating the effect of adding Repatha to statins for reducing cardiovascular risk is ongoing. Repatha is marketed by Amgen Inc., of Thousand Oaks, Calif.