Dive Brief:
- Orphan disease specialist Albireo Pharma on Thursday announced plans for a pivotal study of its lead liver disease drug, following discussions with both the Food and Drug Administration and the European Medicines Agency.
- News of the planned Phase 3 program, expected to begin in the second half of 2017, pushed up the Boston-based biotech's stock up by nearly 5% Friday morning.
- A six-month placebo-controlled Phase 3 trial in children with progressive familial intrahepatic cholestasis (PFIC) will be followed by an open label long-term extension. For the FDA, change in pruritus will be the primary endpoint for evaluation while serum bile acid responder rate will be the primary endpoint for the EMA.
Dive Insight:
Albireo Pharma's planned Phase 3 trial has been designed to build on the data from its Phase 2 study, where its drug — known as A4250 — improved itching and serum bile acid levels in children with a number of cholestatic liver diseases.
PFIC is estimated to affect between one in 50,000 to one in 100,000 people worldwide. There are three different subtypes, PFIC1, 2, and 3, all with separate genetic changes. The disorder triggers progressive liver disease, leading to severe itching, jaundice and stunted growth.
The planned Phase 3 trial is designed with two primary endpoints, one for review by the FDA and the other for the EMA. The endpoint not assessed as primary by each respective regulatory authority will serve as a key secondary endpoint.
This design allows a single trial to be carried out in the U.S., Canada, Europe, the Middle East and Australia — important when working in rare diseases with restricted patient pools.
Albireo is also pooling and analyzing long-term PFIC patient data to support the use of serum bile acid responder rate as an endpoint.
There are no therapeutics specifically approved for PFIC. Current treatment targets the symptoms of cholestasis and pruritus (itching), and include drug therapies or surgical interventions such as partial external biliary diversion or liver transplant. Few other drugs are in development for this rare disease. Shire, for one, has breakthrough therapy designation for its SHP625 (maralixibat) in PFIC2, which is currently in Phase 2 development.