It will immediately begin to sell Bylvay to treat patients impacted by PFIC, a rare cholestatic liver disease that often leads to cirrhosis and liver failure within the first ten years of life.
Bylvay is approved.
Albireo Pharma, a spinout of AstraZeneca, won regulatory approval for the first drug aimed at pruritus in patients with all subtypes of progressive familial intrahepatic cholestasis (PFIC). It is the company’s first drug approval.
The Boston-based company announced that the U.S. Food and Drug Administration (FDA) greenlit ileal bile acid transport inhibitor (IBATi) odevixibat, which will be sold under the brand name Bylvay. It will immediately begin to sell the drug to provide treatment for patients impacted by PFIC, a rare cholestatic liver disease that often leads to cirrhosis and liver failure within the first ten years of life. The sale of Bylvay will be supported by Travere Therapeutics.
Ron Cooper, president and chief executive officer of Albireo, said Bylvay is the first non-surgical treatment that the FDA has approved for PFIC. Prior to the approval of Bylvay, treatment options for PFIC included invasive surgical procedures, such as biliary diversion surgery or liver transplant.
Albireo CEO thanked the patients and families who participated in the decade-long research of Bylvay and expressed his hope that the medication will significantly benefit future patients. There are an estimated 100,000 patients with cholestatic liver disease for which there is not an approved drug. Of those patients, approximately 15,000 are estimated to have PFIC, however, Albireo said those numbers do not include potential patients in India and China.
About Bylvay Approvals
Bylvay was approved based on data from two Phase III trials, PEDFIC 1 and PEDFIC 2. In PEDFIC 1, Bylvay demonstrated statistically significant reductions in serum bile acids (sBAs), as well as improvements in pruritus in pediatric PFIC patients. Long-term data from the PEDFIC 2 study showed the medication provided durable and continued reductions in sBAs, as well as improvements in pruritus assessments. The data also showed improved growth function in patients treated up to 48 weeks with Bylvay.
In addition to the approval by the FDA, Bylvay was also authorized for use in the European Union for all PFIC subtypes. Like in the U.S., Albireo plans to market Bylvay across Europe immediately.
PFIC is caused by an impaired bile flow due to genetic defects. Signature symptoms of PFIC and other cholestatic liver diseases include the intense itching known as pruritus.
In a previous interview with BioSpace, Cooper described the itch as something that “can’t be scratched.” He said the patients would “severely claw” themselves to reach the source of the itch, all to no avail.
Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 and PEDFIC 2, noted the difficulties of treating pediatric PFIC patients. With the approval of Bylvay, Thompson said these patients have a non-surgical option that will change how these patients can be treated.
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,” Thompson said in a statement.
With the approval of Bylvay, Albireo was granted a Rare Pediatric Disease Priority Review Voucher (PRV), which the Company plans to monetize.
Albireo is also studying the use of Bylvay in other rare pediatric cholestatic liver diseases. In the BOLD Phase II clinical trial, the drug is being assessed in patients with biliary atresia, and in the ASSERT Phase 3 clinical trial, Bylvay is being studied in Alagille syndrome. Topline data from the ASSERT trial is expected in 2022, and the BOLD trial data is expected in 2024.
