Cambridge biotech Aegerion wins FDA approval for drug to treat rare form of high cholesterol

Aegerion Pharmaceuticals said Monday it has won approval from the Food and Drug Administration to market the Cambridge biotechnology company’s first drug, a treatment for a rare inherited genetic disorder that can raise cholesterol to life-threatening levels.

The condition, disordehomozygous familial hypercholesterolemia—or HoFH—is resistant to statins and other medications typically used to bring high cholesterol under control.

“We are excited that Juxtapid will become a new treatment option for patients with HoFH,” said Aegerion chief executive Marc Beer. “The approval of our first product also marks an important corporate milestone for Aegerion and reflects our commitment to help patients in need.”

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The drug will be sold in pill form nationwide and taken by patients in conjunction with a low-fat diet and other lipid-lowering treatments. In a 78-week clinical trial, the drug reduced patients’ LDL (low-density lipoprotein) cholesterol levels by an average of 50 percent.

Katherine Wilemon, president and founder of the FH Foundation, a nonprofit patient advocacy group based in South Pasadena, Calif., said the new drug’s approval marks a significant change in the way the genetic disorder is treated.

It’s “a major step forward for HoFH patients and their families, who have long been waiting for new therapies,” Wilemon said. “New treatments, combined with further understanding and awareness of this disease, can bring much needed hope to the HoFH community.”

Juxtapid does carry a risk of liver toxicity, however, so the drug will come with a so-called “black box” label—the FDA’s most serious warning level.

As a result of those health concerns, it will initially be available through a restricted program in which patients are closely monitored by their doctors and pharmacists. The FDA also included as a condition of approval the requirement that Aegerion conduct a follow-up study of the drug’s long-term safety and effectiveness.

Regulators did not sign off use of the drug in children or patients with other types of high cholesterol.