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FDA approval for Bylvay, the first drug treatment for patients With progressive familial intrahepatic cholestasis.

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Last updated:24th Sep 2021
Published:21st Jul 2021
-Albireo Pharma, Inc. announced FDA approval of Bylvay (odevixibat), the first drug approved for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC). Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), which does not require refrigeration and is easily administered as a once-daily capsule or opened and sprinkled onto soft foods.

Albireo is launching Bylvay immediately to accelerate availability for the patients and families impacted by PFIC. “Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a non-surgical option and will change how we treat PFIC,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 and PEDFIC 2. “With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed and we are hopeful for better outcomes for these children.”

PFIC is a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. The most prominent and problematic ongoing manifestation of PFIC is pruritus, or intense itching, which often results in a severely diminished quality of life. Until now, there have been no approved drugs for PFIC. Only surgical options that include biliary diversion surgery (BDS) and liver transplantation have been available, and without them, most PFIC patients do not survive past the age of 30. There are an estimated 100,000 patients with cholestatic liver disease without an approved drug treatment. Of those patients, there are approximately 15,000 with PFIC (excluding China and India).

Condition: Progressive Familial Intrahepatic Cholestasis
Type: drug