Albireo's rare liver disease drug Bylvay scores back-to-back approvals in U.S., Europe

With a pair of approvals on both sides of the Atlantic, liver disease-focused biotech Albireo is off and running with its new medicine Bylvay.

The oral drug, the first treatment for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC), scored an FDA nod Tuesday and a European approval Monday. With the FDA's blessing, Albireo is kicking off an "immediate" launch.

Analysts with Evaluate Pharma have predicted the drug could generate nearly $300 million in PFIC by 2026. Albireo, an AstraZeneca spinoff, is also testing the drug in biliary atresia and Alagille syndrome.

PFIC is a rare, progressive and life-threatening disease that starts early in childhood and affects many thousands of patients worldwide. Most patients suffer from cirrhosis and liver failure within 10 years of life. 

Pruritus, or intense itching, is the most common manifestation of the disease and results in a "severely diminished quality of life," Albireo says.

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Other PFIC treatment approaches include surgical procedures such as a liver transplant. Without them, many patients don't live past the age of 30.

Albireo estimates that 100,000 patients worldwide suffer from cholestatic liver disease without an approved treatment. Of them, more than 15,000 have PFIC.

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The FDA approved Bylvay based on phase 3 trials showing the drug can help ease pruritus and reduce patients' serum bile acids. It isn't yet known whether it can reduce the need for liver transplants.

Along with the Bylvay approval, Albireo also scored a Rare Pediatric Disease Priority Review Voucher. Such vouchers can be used to speed up drug reviews at the agency and can fetch a pretty penny on the resale market.