Rare Liver Disease Drug by Boston Biotech to Cost $385,000 every year

Albireo Pharma, a Boston startup, announced on Wednesday that its new therapy for an ultrarare Liver illness would have an eye-popping average list price of $385,000 per year, only one day after receiving its first US prescription approval. Bylvay is the first pharmaceutical approved to treat progressive familial intrahepatic cholestasis or PFIC. According to Ron Cooper, chief executive and president of Albireo, this severe hereditary illness often leads to cirrhosis and Liver failure in the first ten years of life.

Without Liver surgery or a transplant, most patients do not live past the age of 30. PFIC affects an estimated 15,000 people globally, including 600 children in the United States, according to Cooper. Due to a mutation, bile builds up in Liver cells, causing pruritus, which causes many children to scratch their skin until it bleeds.

The condition has a financial impact on families that may outweigh cystic fibrosis, another rare disease that affects children in terms of missed employment and lost pay. In addition, Bylvay is available as a daily capsule to be ingested or opened and sprinkled on soft meals. According to Cooper, the list price reflects the medicine’s efficacy in a late-stage worldwide clinical trial involving 62 patients and the fact that it is the first licensed treatment for the disease.

The medication was approved for use in Europe by the European Commission on Monday. Cedar Ventura, Emily Ventura’s 9-year-old daughter, was diagnosed with the disease at the age of 5 months and had a Liver transplant four years ago, according to Emily Ventura, executive director of the nonprofit PFIC Network. Even though Cedar is too old to benefit from the treatment, Ventura, who lives in rural Kentucky, believes Bylvay gives many families throughout the world hope.