- Thinly traded micro cap Albireo Pharma (NASDAQ:ALBO) is up 7% premarket on light volume on the heels of its announcement that the FDA has granted Rare Pediatric Disease Designation for lead drug A4250 for the treatment of a rare life-threatening liver disease called progressive familial intrahepatic cholestasis (PFIC), an Orphan Drug indication in the U.S. and Europe.
- If A4250 is approved, the company will receive a Rare Pediatric Disease Priority Review Voucher that it can use for accelerated review for a future marketing application or it can sell it to a third party.
- A4250, an ileal bile acid transporter inhibitor, is currently in Phase 3 development for PFIC.