AstraZeneca spinout Albireo Pharma is moving closer to securing regulatory approval for its treatment known as odevixibat, for pruritus in patients with PFIC.
Odevixibat is close to approval.
AstraZeneca spinout Albireo Pharma is moving closer to securing regulatory approval for its treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
This morning, the company announced the U.S. Food and Drug Administration accepted the company’s New Drug Application for Albireo Pharma’s ileal bile acid transport inhibitor (IBATi) odevixibat. The FDA granted Priority Review to odevixibat and set a Prescription Drug User Fee Act (PDUFA) goal date of July 20. Odevixibat previously received Fast Track, Rare Pediatric Disease and Orphan Drug Designations in the U.S.
The FDA accepted the NDA based on Phase III data from the PEDFIC 1 study, the first Phase III study conducted in PFIC. In that study, odevixibat demonstrated statistically significant reductions in serum bile acids (sBAs) and improvements in pruritus in pediatric PFIC patients. Additionally, long-term data from PEDFIC 2, an ongoing open-label Phase III extension study, demonstrate continued and durable reductions in sBAs, improvements in pruritus assessments and encouraging markers of liver and growth function in patients treated up to 48 weeks with odevixibat.
Ron Cooper, president and chief executive officer of Albireo Pharma, is excited about the potential for odevixibat, which could become the first approved drug treatment for patients with PFIC, a cholestatic liver disease caused by an impaired bile flow due to genetic defects. The bile build-up in liver cells causes the liver disease. The signature symptom of PFIC and cholestatic liver disease is pruritus, an intense itching. The itch is something that can’t be scratched and Cooper said patients will severely claw at themselves as they attempt to reach the source. There are no drugs approved to treat PFIC, which can lead to cirrhosis and liver failure. Currently the only options for this disease are off-label uses of some approved medications, such as ursodeoxycholic acid, or invasive surgical procedures, such as partial external biliary diversion and liver transplants.
The company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021 if approved by the FDA.
In a previous interview with BioSpace, Cooper said the company has been working toward this moment for the past 10 years. Not only could odevixibat benefit PFIC patients, the company is assessing the drug in other cholestatic diseases like biliary atresia and Alagille syndrome. If odevixibat pans out in other ongoing studies, Cooper speculated the drug could become a pipeline in a product. Odevixibat is currently being evaluated in the ongoing PEDFIC 2 open-label trial, the BOLD Phase III trial in patients with biliary atresia, and the global Phase III ASSERT trial for ALGS.
“With no approved treatments, PFIC presents an exciting commercial opportunity and will pave the way for expected additional indications in Alagille syndrome and biliary atresia,” Cooper said in a statement this morning.
In addition to filing a regulatory submission in the United States, the company is also seeking approval in Europe. The European Medicines Agency has also accepted Albireo’s regulatory submission. Odevixibat is the only IBATi granted accelerated assessment by the EMA. It has also been granted Orphan Designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC.
