The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of Alagille syndrome, a rare and life-threatening disease that affects the liver and has no approved pharmacologic treatment option.
A4250 also holds Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Alagille, and Orphan Drug designations from both the FDA and EMA for the treatment of progressive intrahepatic cholestasis (PFIC). The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and provides 7 years of market exclusivity for the product upon regulatory approval.
“We are very pleased with the FDA´s designation of A4250 as an orphan drug for the treatment of Alagille syndrome,” said Ron Cooper, President and Chief Executive Officer of Albireo. “While we are focused on executing our PEDFIC-1 Phase 3 clinical trial of A4250 in PFIC, there is extremely high unmet need across numerous rare cholestatic liver diseases, including Alagille. This further highlights the development potential of A4250.”
Alagille syndrome is a genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. Liver damage caused by cholestasis (bile flow blockage) is a major feature of the disease. Bile ducts may be narrow, malformed or fewer in number. As a result, bile builds up in the liver and causes scarring. Signs and symptoms may include jaundice, poor weight gain and growth, and severe pruritus (itching), and generally become evident in infancy or early childhood. Symptoms range from mild to severe, sometimes requiring transplantation. Alagille is estimated to affect between one in every 30,000 to 70,000 children born worldwide.
About A4250
A4250 is a first-in-class product candidate being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis (PFIC). A highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), A4250 has minimal systemic exposure and acts locally in the gut.
The PFIC A4250 program or elements of it have received Fast Track, Rare Pediatric Disease and Orphan Drug designations in the United States. In addition, the FDA has granted Orphan Drug Designation to A4250 for the treatment of Alagille syndrome. The European Medicines Agency (EMA) has granted A4250 Orphan Drug Designation, as well as access to the PRIority MEdicines (PRIME) program for the treatment of PFIC, and its Paediatric Committee has agreed to Albireo’s A4250 Pediatric Investigation Plan. A4250 is currently being evaluated in a Phase 3 clinical program in patients with PFIC, subtype 1 or 2 (NCT03566238).
