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Praluent – FDA approved as Cholesterol-lowering Medicine for Patient non responsive to Statin due to Genetic origin of Hypercholesterolemia

Reporter: Aviva Lev-Ari, PhD, RN

The first of a powerful new class of cholesterol-lowering medicines won approval from U.S. regulators Friday—a highly anticipated medical advance that nevertheless promises to escalate the growing clamor over drug costs.

The drug, called Praluent and developed by Regeneron Pharmaceuticals Inc. and SanofiSA, provides a new and in some cases desperately needed option for several million high-risk heart patients who can’t get their cholesterol to desirable levels with the blockbuster group of medicines known as statins.

• Praluent works by blocking a protein called PCSK9, which interferes with the body’s ability to clear artery-damaging cholesterol from the blood.
• A series of genetic discoveries a decade ago laid the foundation for Praluent and its rivals. After French researchers linked mutations in the PCSK9 gene to high LDL levels and early heart disease in French families, researchers Helen Hobbs and Jonathan Cohen at University of Texas Southwestern Medical Center, Dallas, wondered if other mutations might have the opposite effect.

But the companies are pricing the drug at $14,600 a year, an especially high amount for a medicine aimed at a common condition like heart disease. By contrast, statins, which are available in generic versions and remain the mainstay drug option for cholesterol reduction, can be purchased for just a few dollars a month.

Regeneron and Sanofi defended Praluent’s price. An antibody that patients inject themselves, it will cost substantially less than similarly administered drugs such as Humira and Enbrel for rheumatoid arthritis and other autoimmune diseases, which are prescribed for thousands of patients and which they said list for more than $38,000 a year.

The toll in the U.S. for cardiovascular disease amounts to more than $300 billion a year, according to estimates, while treating an individual heart attack can range from $60,000 to $120,000, said Leonard Schleifer, Regeneron’s chief executive officer. Expectations—not yet proven—are that the marked cholesterol reductions seen with Praluent will translate into fewer deaths and costly events.

The company believes “we’ve come up with a price that provides value to the health-care system,” Dr. Schleifer said.

• Regeneron says it believes between 8 million and 10 million patients in the U.S. meet those criteria.
• Statins are taken by some 40 million Americans, and in addition to a healthy diet and exercise, remain the mainstay strategy to lower LDL cholesterol, the chief culprit in the accumulation of deposits in the coronary arteries that lead to heart attacks.

SOURCE

http://www.wsj.com/articles/fda-approves-cholesterol-drug-from-regeneron-sanofi-1437762374