FDA Grants Rare Pediatric Disease Designation to Albireo’s Candidate for Rare Liver Disease

Rare Daily Staff

The U.S. Food and Drug Administration granted Albireo Pharma’s lead candidate A4250 Rare Pediatric Disease designation for the treatment of progressive familial intrahepatic cholestasis, a rare and life-threatening liver disease with no approved drugs.

PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease. Moderate to severe pruritus, an itching of the skin, is symptom of the condition that impairs a patient’s quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. There are no approved medicines for PFIC.

A4250 is a first-in-class experimental therapy being developed to treat rare pediatric cholestatic liver diseases and is in phase 3 development in PFIC.

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old and affect fewer than 200,000 persons in the United States. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

If A4250 is approved by the FDA for PFIC, the Rare Pediatric Disease designation may enable Albireo to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future drug approval submission, which would reduce the FDA review time from ten months to six months.

The vouchers are potentially lucrative because they are transferable. Most recently, Spark Therapeutics sold a priority review voucher at the end of April for $110 million.

“This designation affirms Albireo’s eligibility to apply for a rare pediatric disease priority review voucher upon submission of a new drug application for A4250 and highlights the serious, life-threatening manifestations of PFIC,” said Ron Cooper, president and CEO of Albireo. “A priority review voucher is a very valuable and important component of the incentives to develop products for rare, life-threatening diseases.”

In addition to the rare pediatric disease designation, A4250 received orphan drug designation for PFIC in the U.S. and E.U., and Albireo was granted access in 2016 to the EMA’s PRIority MEdicines (PRIME) program for the treatment of PFIC.

June 13, 2018
Photo: Ron Cooper, president and CEO of Albireo