FDA issues rare pediatric disease designation for Albireo Pharma's drug A4250

PFIC is a sexual, life-threatening liver disease that affects between 50,000 and 100,000 newborns worldwidemoderate to severe itching is a common clinical problem in PFIC, which significantly reduces quality of lifeIn many cases, PFIC can lead to cirrhosis and liver failure within the first 10 years, and almost all PFIC patients need treatment before the age of 30There is currently no approved PFICdrug(treatment regimen)recently, Albireo Pharma announced theof the U.SFDA (a rare paediatric disease designation for thecompany's(the leading candidate drug, A4250, for the treatment of experimental family-borne hepatic bile siltation (PFIC)a strong selective inhibitor of the A4250
A4250 is a recinoic acid transport protein (IBAT) with very small systemic exposure that can function locally in the intestineThis potential "first-in-class" candidate for this indication is under going into clinical development in Phase 3in addition to this qualification in the United States, the EuropeanMedicines(EMA) has also issued orphan drug eligibility for The A4250 treatment PFIC, as well as the Priority Drug Qualification (PRIME) The EMA Paediatric Committee has approved the A4250's paediatric research program Albireo's single, randomized, double-blind, placebo-controlled phase 3 clinical trial (the first patient has been raised) The clinical trial was designed to assess the efficacy of the drug in 60 patients with elevated serum bile acid (sBA) levels and itching type 1 or type 2 PFIC at age 6 months to 18 years of age If successful, data from the Phase 3 trial and open label expansion study will form the basis for the A4250's approval in the Us and the European Union