The FDA approved alirocumab (Praluent; Sanofi and Regeneron Pharmaceuticals) for patients with heterozygous familial hypercholesterolemia (HeFH) and for patients with clinical atherosclerotic cardiovascular disease, according to a press release from the FDA July 24.
HeFH is an inherited disease that causes high levels of low-density lipoprotein (LDL) cholesterol in patients. Alirocumab is designed to lower this “bad” cholesterol and is an injectable monoclonal antibody and is the first in its class of PCSK9 inhibitors.
"This new class of medications can significantly and effectively lower LDL cholesterol in very high-risk patients who could not otherwise achieve acceptable LDL cholesterol levels,” said MUSC Health cardiologist Pamela B. Morris, M.D., who is the principal investigator for multiple trials of PCSK9 inhibitors at MUSC. “Long-term outcomes trials in progress will determine if this dramatic reduction in LDL-C results in a reduction in cardiovascular events," says Morris. (To learn more about PCSK9 inhibitors, their mechanism of action, and clinical trials with these inhibitors at MUSC, click here.)
This new drug is most successful when combined with a healthy diet and maximum-tolerated statin therapy. It is not indicated, however, for statin-intolerant patients without established clinical atherosclerotic cardiovascular disease.
Clinical trials for this drug in children have not yet been performed, so it is only prescribed to patients 12 years of age and older.
The side effects of this drug include itching, swelling, pain, or bruising at the injection site, nasopharyngitis, and the flu. There have also been reported cases of allergic reactions to this drug, causing hypersensitivity vasculitis and hypersensitivity reactions requiring hospitalization.
It is expected the FDA will approve a second PCSK9 inhibitor called evolocumab (Repatha; Amgen) by August 27.