By David Krechevsky, Waterbury Republican-American, Conn.
Mar. 27–A Cheshire, Conn., pharmaceutical company has set the price for a new drug to treat a rare blood disorder at more than $389,000 a year. Despite the hefty price for the new drug, trade-named Soliris, Alexion Pharmaceuticals Inc. is determined to make the drug available to anyone who needs it. It plans to help needy patients find financial aid, either from insurance companies or other sources, and is setting up its own charitable foundation.
Company officials took part in a conference call and audio Webcast with shareholders and corporate analysts Monday morning to discuss the launch of Soliris, which the U.S. Food and Drug Administration approved earlier this month.
Soliris, or eculizumab, treats a rare blood disorder called paroxysmal nocturnal hemoglobinuria, or PNH, which can lead to disability and premature death. The first vials of Soliris are expected to be shipped next week, officials said.
Both the FDA and the European Agency for the Evaluation of Medicinal Products have designated Soliris an “orphan” drug. Under the Orphan Drug Act of 1983, companies that develop drugs for rare disorders are eligible for tax cuts on research costs. They also retain exclusive marketing rights to their product for seven years in the United States and 10 years in the European Union following its approval, according to the FDA.
Alexion officials estimate that just 8,000 to 10,000 people in North America and Europe are afflicted with PNH. “Our goal is that every patient who can benefit from Soliris will have access to Soliris,” David Hallal, vice president of U.S. commercial operations, said during the Webcast.
To accomplish that, the company will assign a registered nurse as a case manager for each patient seeking the drug. The nurses, who will be based in Connecticut, “have extensive insurance and health care industry expertise” and will help patients find financial aid. In addition to the case managers, Alexion has formed a nonprofit corporation, the Alexion Complement Foundation, a charity for qualified patients who don’t have insurance.
David W. Keiser, president and chief operating officer, said Alexion set the wholesale price at nearly $5,000 per vial based on a number of factors, including the rarity of the disease, development and production costs, and “the cost to sustain the commitment to the PNH community.” The price is comparable to other orphan drugs, he said, citing the $400,000 price for myozyne, which treats Pompe disease, an often fatal disorder of the heart; and the more than $800,000 price for elaprase, which treats Hunter’s Syndrome, a rare genetic disorder. Abbey S. Meyers, president of the Danbury-based National Organization for Rare Disorders Inc., said high prices often are necessary for orphan drugs.
“If a company is going to develop a drug for high blood pressure or some other common disease, they can put a little bit of profit on each pill and make a lot of money,” she said. “If they make a medicine for a limited number of people, they wouldn’t be able to make a profit doing that.”
Companies need to make a profit to recover their development costs and to support research on other drugs, she said. “It will be some years before Alexion can make a profit” on Soliris, Meyers said. The drug was in development for 15 years, and the company has yet to post a profitable quarter.
Keiser said Alexion is aware the price is steep, but is doing what it can to help.
“When you have a therapy that in fact has very compelling clinical benefits for those patients, in our view it’s our obligation to bring that therapy to those patients,” he said.
SOLIRIS AT A GLANCE
–Manufacturer: Alexion Pharmaceuticals Inc., Cheshire
–Vial size: 300 milligrams
–Vial price: $4,992 (wholesale)
–Dosage: 900 milligrams, 26 treatments per year
–Dosage price: $389,376 (wholesale)
ON THE WEB:
–Alexion: www.alexionpharm.com
–National Organization for Rare Disorders Inc.: www.rarediseases.org
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ALXN,
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