Albireo Pharma is in the midst of preparing for an exciting Phase 3 clinical trial to test A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC).
PFIC is a rare group of pediatric liver conditions that affect the transportation of bile out of the liver. To learn more information about PFIC, click here.
A4250 is a potent inhibitor of the ileal bile acid transporter, so the drug would work to target the underlying problem behind PFIC, a huge improvement over the current treatment options for this debilitating condition.
Presently, the only treatment options are symptom management or intense surgeries. Since the condition affects mostly children, there is a great need for a pharmaceutical treatment option. Ideally, A4260 will help not only lower serum bile acid levels, but also improve pruritus, which is an intense itching as a result of the condition.
This movement towards an A4250 approval is a result of the prior Phase 2 studies proving that A4250 was well-tolerated in patients. The Phase 3 trials will have sites all over the world, with enrollment to begin in the second half of this year. The company has also undergone consultations with the U.S. FDA, with the hope that the results from the trials will support a subsequent approval of the drug.
If you want to learn more about A4250 and this study before enrollment in the trials begin, click here, and keep a look out for more information from Albireo and the PFIC community!