Benzinga | Jul 20, 2020 08:03AM EDT

Albireo Pharma Announces the Launch of an Expanded Access Program for its Odevixibat

Albireo Pharma, Inc. (NASDAQ:ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the launch of an Expanded Access Program (EAP) for investigational lead product candidate odevixibat, a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), for patients with progressive familial intrahepatic cholestasis (PFIC) in the U.S., Canada, Australia and Europe.

"A tremendous need remains for a pharmacologic treatment option for PFIC, a life-threatening, ultra-rare, pediatric liver disease with serious and debilitating symptoms. Through our open-label PEDFIC 2 trial, and now our Expanded Access Program (EAP), we hope to increase access to odevixibat for all eligible patients to the greatest extent possible until odevixibat is commercially available," said Ron Cooper, President and Chief Executive Officer of Albireo. "We are pleased to launch the program in the U.S., Canada, Australia and Europe, and we are working closely with local regulators in other countries to offer access and continue to provide hope for PFIC families worldwide."

Odevixibat is being evaluated in patients with PFIC in the PEDFIC 1 Phase 3 trial (NCT03566238), for which topline results are expected in mid-2020, and the PEDFIC 2 open-label trial (NCT03659916) evaluating the long-term efficacy and safety of odevixibat in patients with PFIC. The PEDFIC 2 trial is the preferred route of access for all patients who qualify, as determined by their physician. Cohort 1 of the trial allows patients from the PEDFIC 1 clinical trial to continue treatment with odevixibat until it is commercially available. Cohort 2 comprises patients who did not participate in the PEDFIC 1 trial and includes other forms of PFIC not included in PEDFIC 1.

The EAP is available to patients in the U.S., Canada, Australia and Europe but may be limited depending on drug supply. Timing of availability in Europe will vary due to country-specific and local regulations. This program is available for patients with a clinical diagnosis of PFIC who have no other therapeutic options and do not qualify for, or have access to, PEDFIC 2 Cohort 2. The EAP inclusion criteria allow participation by patients with reduced pruritus and serum bile acid levels compared to our existing studies. All forms of PFIC (even episodic forms -- e.g., benign recurrent intrahepatic cholestasis, commonly known as BRIC) are allowed, genetic confirmation of PFIC is not required, and patients who have had a prior liver transplant may be eligible for this program, as determined by their physician. For more information on Albireo's odevixibat Expanded Access Program, visit www.albireopharma.com/patients-families/expanded-access-policy.

If you are a physician in the U.S. or Canada who would like to request EAP access for your patient, contact Odevixibat@clinigengroup.com. If you are a physician in Australia or Europe who would like to request EAP access for your patient, contact medicineaccess@clinigengroup.com.

Our aim is to expand this program to other geographies over time. If you are a physician, patient or caregiver of a patient outside of the U.S., Canada, Australia, or Europe who may be interested in odevixibat therapy access, email medinfo@albireopharma.com with your interest.