On the heels of launching Bylvay as the first treatment for rare pediatric liver disease progressive familial intrahepatic cholestasis (PFIC), Albireo has presented data that show the drug is similarly effective against another pediatric liver disorder.
A phase 3 trial of Bylvay (odevixibat) on patients with Alagille syndrome has met its primary objective to improve the unrelenting pruritis (itching) that accompanies the disorder and its secondary goal to reduce serum bile acids. In addition, there were no discontinuations of treatment among the 52 patients who received Bylvay, the primary side effect of which is diarrhea.
Boston-based Albireo will immediately submit filings to the FDA and the European Medicines Agency. Prior discussions with the regulators indicate that a successful single study will be sufficient for approval, the company said.
In July of last year, Albireo’s data in PFIC were compelling enough that the two regulators signed off on Bylvay four days apart.
“They were similarly spectacular, to be honest,” Albireo CEO Ron Cooper said in an interview, comparing the results between the PFIC and Alagille trials. “We’re going in with gold-standard study, with pretty unequivocal results.”
An approval to treat Alagille syndrome would roughly double the potential patient population for Bylvay. It is estimated that approximately 25,000 people worldwide have each condition.
Albireo also is investigating Bylvay to treat a similar but more common pediatric liver disease called biliary atresia. The company is enrolling patients in a phase 3 study that's scheduled to read out in 2024. If the company can score endorsements in all three of the diseases, it sees blockbuster potential for Bylvay.
Alagille syndrome and PFIC are inherited conditions that cause a buildup of bile in the liver. Life expectancy is shorter with PFIC, with only 50% making it past age 10. For many with either condition, a liver transplant becomes necessary.
“A high percentage of Alagille patients get a liver transplant because of pruritis,” said Cooper, who added that he is hopeful that Bylvay could help both PFIC and Alagille patients eventually avoid those procedures.
Once-daily oral treatment Bylvay is an ileal bile acid transporter (iBAT) that acts in the small intestine, reducing reuptake of bile acids and increasing clearance of bile acids through the colon.
Also from the iBAT drug class is Mirum Pharmaceuticals’ Livmarli, which won approval to treat Alagille syndrome last year and is nearing completion of a phase 3 study in PFIC. In the second quarter, Livmarli generated sales of $17.5 million, compared with $5.9 million for Bylvay.
With an approval in Alagille syndrome, however, Albireo could be in position to attract more patients as its drug had a smoother trip through the clinic. Livmarli flunked two phase 2 trials under its previous owner, Shire, before Mirum got it across the FDA finish line.
Albireo also is developing a liver disease drug for adults. That midstage drug, A-3907, is the world’s first oral systemic apical sodium-dependent bile acid transporter candidate.