PFIC Drug Bylvay Has List Price of $385K


It’s no secret that drugs can sometimes be expensive, especially in regards to rare diseases or conditions. But how much would you guess a new drug is priced when first on the market? According to MSN, the list price for Bylvay, the first approved treatment for progressive familial intrahepatic cholestasis (PFIC), sits at $385,000 per year. Although the treatment should be more affordable for patients through insurance and a payment program via Albireo, the initial cost is eye-opening – and shocking – to learn.


In a prior news release to investors, Albireo describes how Bylvay works:

A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay acts locally in the small intestine. Bylvay does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population.

Currently, the recommended Bylvay dose is 40 mcg/kg once daily (in the morning). The treatment should be taken with a meal. Altogether, the treatment is designed for patients with PFIC, a group of rare genetic liver disorders which can lead to liver scarring and failure within the first 10 years of a child’s life. This treatment is extremely important in improving quality of life and overall survival; after all, a majority of patients with PFIC require a liver transplant before age 30.

Currently, the list price sits at $385,000, a hefty sum. Albireo explains that the treatment is listed so high due to a number of factors, including its efficacy in clinical trials and its status at the first approved PFIC treatment. However, it is important to recognize that the list price is not always the final price. Novo-Nordisk explains that the list price is typically only used by those without insurance, those who still need to fulfill deductible requirements, or those within a Medicare Part D coverage gap. For others, their price may depend on rebates, insurance, wholesaler fees, and other elements.

Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive familial intrahepatic cholestasis (PFIC) represents a group of rare inherited liver conditions which can cause progressive liver failure or liver disease. Altogether, there are three forms of PFIC: 1, 2, and 3. Each condition results from a specific genetic mutation which interrupts bile flow throughout the body. In PFIC, patients’ liver cells cannot secrete enough bile, causing bile to accumulate in the liver. Typically, symptoms appear in infancy and often within the first few months of life. However, in rarer cases, patients may not present with symptoms until adolescence. Symptoms include:

  • Jaundice (yellowing of the skin and eyes)
  • Cholestasis (bile obstruction)
  • Pancreatitis
  • Pruritus (intense itching)
  • Fatigue
  • Liver and spleen enlargement
  • Hearing loss
  • Diarrhea
  • Short stature
  • Fat-soluble vitamin deficiencies
    • Note: These may cause broken bones, difficulty balancing, or vision problems.
  • Failure to thrive
  • Suicidal ideation
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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