FDA Grants Rare Pediatric Disease Designation to Albireo’s Liver Disease Drug


The U.S. Food and Drug Administration (FDA) granted Boston-based Albireo Pharma’s A4250 rare pediatric disease designation.

A4250 is an ileal bile acid transporter (IBAT) inhibitor under development to treat progressive familial intrahepatic cholestasis (PFIC). PFIC causes progressive liver disease that generally leads to liver failure. In it, liver cells have limited ability to secrete bile, which builds up in the liver cells. Symptoms are typically seen in infancy, and include severe itching, jaundice, failure to gain weight and grow, high blood pressure in the vein that leads to the liver, and an enlarged liver and spleen.

A4250 is currently in Phase III development for PFIC. The European Medicines Agency (EMA) has also granted it orphan drug designation, as well as access to its PRIority MEdicines (PRIME) program for PFIC. The EMA’s Paediatric Committee has also agreed to Albireo’s A4250 Pediatric Investigation Plan.

The Phase III PEDFIC-1 trial recently launched. It is a single, randomized, double-blind, placebo-controlled Phase III trial. It will evaluate the drug in 60 patients ages six months to 18 years with PFIC, subtype 1 or 2, who have elevated serum bile acid levels and pruritus. If positive, data from the trial and an open-label extension trial will be used to submit the drug to the FDA and EMA.

“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, Albireo’s president and chief executive officer, in a statement. “A priority review voucher is a very valuable and important component of the incentives to develop products for rare, life-threatening diseases.”

Albireo’s focus is on developing and commercializing novel bile acid modulators to treat orphan pediatric liver diseases and other liver or gastrointestinal diseases. The parent company is Albireo Pharma, located in Boston. Its key operating subsidiary is Albireo AB, located in Gothenburg, Sweden. It was spun out from AstraZeneca in 2008.

In May, the company provided a 2018 Business Update. Part of the big news was the company’s elobixibat, which because the first IBAT inhibitor ever approved anywhere. Japan’s Ministry of Health, Labor and Welfare approved elobixibat to treat chronic constipation in Japan in January 2018. This also triggered a milestone payment from EA Pharma of more than $10 million.

The Elobix AB subsidiary entered into an agreement to monetize its royalty rights under its license deal in Japan with HealthCare Royalty Partners, receiving a $45 million payment with an additional $15 million possible.

The company has so far raised $94.1 million in equity financings.

Although different countries define orphan diseases and rare diseases slightly different, the FDA’s rare pediatric disease designation applies to serious or life-threatening disease that mostly affect people from birth to 18 years of age and that affect fewer than 200,000 people in the U.S. The FDA’s Rare Pediatric Disease Priority Review Voucher program allows the company who receives an approval of a new drug application or biologics license application for a drug in that category to receive the voucher, which can be redeemed to obtain priority review for any subsequent marketing application, or can be sold or transferred to another company.

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