Rare Daily Staff

Albireo Pharma, a rare pediatric liver disease company developing novel bile acid modulators, said it has entered into a definitive agreement to sell its Priority Review Voucher for $105 million.

Albireo received the voucher in July with the U.S. Food and Drug Administration approval of Bylvay for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease.

The most prominent and problematic ongoing manifestation of PFIC is pruritus, or intense itching, which often results in a severely diminished quality of life, which until now had no approved therapies.

Bylvay is a potent, non-systemic ileal bile acid transport inhibitor, which does not require refrigeration and is easily administered as a once-daily capsule or opened and sprinkled onto soft foods.

The Priority Review Voucher (PRV) was granted to Albireo under an FDA provision that encourages the development of treatments for rare pediatric diseases. A PRV may be issued to the sponsor of a rare pediatric disease product application that entitles the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval. The sponsor receives the PRV upon approval of the rare pediatric disease product application and it can be sold without limitation.

“The sale of the PRV provides Albireo with an important source of non-dilutive capital and further strengthens our balance sheet,” said Ron Cooper, president and CEO of Albireo. “The proceeds allow us to continue our mission to ensure that all diagnosed children with PFIC in the U.S. and Europe will have access to Bylvay, progress two additional Bylvay phase 3 studies and advance two new product candidates.”

Albireo will receive a payment of $105 million upon the closing of the transaction, which is subject to customary closing conditions and is expected to occur following expiration of the applicable U.S. antitrust clearance requirements.

Photo: Ron Cooper, president and CEO of Albireo.

X