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Boston biotech’s drug for rare liver disease will cost $385,000 a year

Boston biotech Albireo Pharma disclosed that its new medicine for an ultrarare liver disease, Bylvay, will come with an eye-popping average list price of $385,000 a year.
Boston biotech Albireo Pharma disclosed that its new medicine for an ultrarare liver disease, Bylvay, will come with an eye-popping average list price of $385,000 a year.Albireo Pharma

The day after winning its first US drug approval, Boston biotech Albireo Pharma on Wednesday disclosed that its new medicine for an ultrarare liver disease will come with an eye-popping average list price of $385,000 a year.

Ron Cooper, the chief executive and president of Albireo, said Bylvay is the first medicine approved to treat progressive familial intrahepatic cholestasis, or PFIC, a deadly genetic disease that often leads to cirrhosis and liver failure in the first 10 years of life. Most patients do not survive past the age of 30 without liver surgery or a transplant.

An estimated 15,000 people worldwide have PFIC, including about 600 children in the United States, Cooper said. A mutation causes bile to build up in liver cells and leads to pruritus, intense itching that makes many children scratch their skin until they bleed. The disease takes a huge financial toll on families in missed work and lost wages that may exceed that of cystic fibrosis, another rare disease that afflicts children.

Bylvay comes in a daily capsule that can be swallowed or opened and sprinkled onto soft foods. The list price reflects how effective the medicine was in a late-stage international clinical trial on 62 patients and the fact that it is the first approved medication for the disease, Cooper said.

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“Up until today, these patients really did not have an option,” he said. Most families that get the drug will have “minimal out-of-pocket costs,” said Cooper, who is optimistic health insurers will cover it and said the company has launched a program to help patients afford the treatment.

The European Commission approved the drug for use in Europe on Monday.

Emily Ventura, executive director of the nonprofit PFIC Network, said her 9-year-old daughter, Cedar Ventura, was diagnosed with the disease at 5 months and underwent a liver transplant four years ago. Although it’s too late for Cedar to benefit from the drug, Ventura, who lives in rural Kentucky, said Bylvay “brings a lot of hope to many families all over the world.”

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Despite its six-figure price tag, Bylvay won’t even be among the ten most expensive medicines in the United States, according to GoodRx, a Santa Monica, Calif., firm that tracks drug prices. The most costly medication is Zolgensma, a one-time gene therapy for spinal muscular atrophy sold by a Novartis subsidiary for $2.1 million.

Albireo was founded in 2008 as a spinout from the British-Swedish drug giant AstraZeneca and went public on the Nasdaq stock market in 2016. The company has more than 130 employees worldwide, most of them located in Post Office Square in Boston.


Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.