Rare Daily Staff

The U.S. Food and Drug Administration approved Albireo Pharma’s Bylvay, the first drug approved for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis.

Progressive familial intrahepatic cholestasis (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. The only available treatment options have been surgical, which include biliary diversion surgery (BDS) and liver transplantation. 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).

Bylvay is a potent, non-systemic ileal bile acid transport inhibitor, 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.”

The approval of Bylvay was supported by data from PEDFIC 1 and PEDFIC 2, the largest, global, phase 3 trials ever conducted in PFIC. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus and serum bile acid primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5 percent of treated patients vs. 5.0 percent of placebo patients).

PEDFIC 2, a long-term, open-label phase 3 extension study, reaffirmed Bylvay delivered sustained reductions in serum bile acids as well as improvements in pruritus assessments, growth and other markers of liver function in patients treated up to 48 weeks. Across both studies, Bylvay was well tolerated with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. There were no serious treatment-related adverse events.

“Until now invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” said Emily Ventura, leader of PFIC Advocacy and Resource Network and mother to a PFIC patient. “As a community, we experience extreme challenges and diminished quality of life for children and families with PFIC. Managing the symptoms can be extremely difficult—the burden is unimaginable with our kids suffering physically, emotionally and developmentally.”

Photo: Emily Ventura, leader of PFIC Advocacy and Resource Network

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