Aegerion Pharmaceuticals said Monday that it has won Food and Drug Administration approval to sell its first drug, a treatment for a rare inherited genetic disorder that can raise cholesterol to life-threatening levels.
The condition, homozygous familial hypercholesterolemia, or HoFH, is resistant to statins and other medications typically used to bring high cholesterol under control.
It affects just 1 in 1 million people, according to the National Institutes of Health.
“We are excited that Juxtapid will become a new treatment option for patients with HoFH,” the Cambridge biotechnology company’s chief executive, Marc Beer, said Monday.
“The approval of our first product also marks an important corporate milestone for Aegerion and reflects our commitment to help patients in need.”
The drug will be available in pill form nationwide beginning in January and will be 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 FH Foundation, a nonprofit patient advocacy group, said the drug’s approval marks a significant change in the way the 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 carries a risk of liver toxicity, however, so the drug will come with a “black box” label — the FDA’s most serious warning level.
As a result of those 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 didn’t approve use of the treatment in children or patients with other types of high cholesterol.
Aegerion has been developing Juxtapid since the company’s inception in 2005, racking up a $171 million operating deficit in that time, according to its most recent quarterly report filed with the Securities and Exchange Commission.
Partly because the potential pool of patients is so small, Juxtapid will be extremely costly, between $200,000 and $300,000 per patient per year.
During a conference call with investors Monday, Beer said patients should expect to wait three to four months from the time they receive a prescription to when they get their first dose of Juxtapid. He attributed the delay to the process of gaining payment authorization from insurers and the need for liver tests and drug education.
Beer declined to provide revenue projections for the drug — while promising to do so at the JP Morgan Health Care Conference in San Francisco next month — but said the company is targeting 3,000 potential Juxtapid users in the US. Aegerion also is seeking approval for the drug in Europe and expects a decision from the regulator there by mid-2013.
The company’s stock price declined 1.8 percent following Monday’s news, which had been anticipated. But Aegerion stock has risen by more than 80 percent since October, when an FDA advisory panel recommended Juxtapid’s approval. The stock closed Monday at $25.25 on Nasdaq. Callum Borchers can be reached at email@example.com. Follow him on Twitter @callumborchers.