Albireo Receives FDA Fast Track Designation for A4250

Albireo Pharma (Nasdaq:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to lead product candidate A4250, an ileal bile acid transporter inhibitor, which is being developed for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare and life-threatening liver disease with no approved pharmacologic treatment option.

As quoted in the press release:

In addition to Fast Track designation, A4250 has received several other special regulatory designations for development in PFIC. The FDA has granted A4250 Orphan Drug Designation and Rare Pediatric Disease designation with eligibility to apply for a Priority Review Voucher. The European Medicines Agency (EMA) has granted A4250 Orphan Drug Designation, as well as access to the PRIority MEdicines (PRIME) program, and its Paediatric Committee has agreed to Albireo’s A4250 Pediatric Investigation Plan.

“Fast Track designation is a recognition of the serious nature of PFIC and the critical unmet medical need, and it reinforces the potential of A4250 to be the first approved pharmacological treatment,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We are making good progress enrolling patients into PEDFIC-1, our A4250 Phase 3 trial, and Fast Track will be helpful in the submission and approval process.”

Click here to read the full press release.