Reimbursed Access to Bylvay® (odevixibat) Approved in France for Patients with PFIC 1 & 2

BOSTON, Nov. 30, 2022 (GLOBE NEWSWIRE) — Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced that the Committee Economic Health Products (CEPS) has approved reimbursed access to Bylvay (odevixibat) in France following a favorable clinical assessment from the Haute Autorité de Santé (HAS) for the treatment of progressive familial intrahepatic cholestasis (PFIC) types 1 and 2 (except for the BSEP3 subtype). A potent, oral, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and acts locally in the small intestine as a treatment for PFIC, a rare and devastating disorder that affects young children and causes progressive, life-threatening liver disease. Bylvay received from the HAS Transparency Committee an Important SMR for its medical benefit and a Moderate ASMR III for its improvement in medical benefit.

“A non-surgical option can offer relief to children suffering from the severe itching that disrupts basic daily activities including sleep, poor attention at school, and reduced quality of life. Having Bylvay as a treatment option could significantly improve how we manage the disease with our patients and may have the potential to modify the progression of the disease,” said Prof. Emmanuel Gonzales, Head of Coordinating Center of Reference Centers for Rare Diseases in Biliary Atresia and Genetic Cholestasis in France.

Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes life-threatening liver disease and symptoms such as intense itching, poor sleep, delayed growth, and diminished quality of life. The harmful impacts of the disease extend beyond the individuals with PFIC to those caring for them as shown by the 2022 multinational PICTURE study, which revealed PFIC negatively affects caregivers’ quality of life, relationships, and career prospects.

“Bylvay’s reimbursement access in France will play an important role in the lives of patients and families who are dealing with the devasting impacts of PFIC,” said Laure Dorey, Executive Director of Association Maladies Foie Enfants (Children’s Liver Disease Association) in France. “A non-surgical option will help young patients who are struggling with daily activities due to the constant itching and other symptoms the disease brings. I’m so thankful to Albireo for their continued efforts to expand the reimbursed access to this treatment option, helping the patients and families that need it most.”

“We are grateful for this favorable decision and for recognizing the clinical and economic value that Bylvay provides to the French PFIC community,” said Steve Arnold, President Global Markets at Albireo. “We are very excited to continue to invest in France and raise awareness of pediatric cholestatic diseases. We are committed to the patients and families impacted by the burden of these devastating diseases in France and globally.”

This availability marks the sixth reimbursement approval for Bylvay in Europe, following launches in Germany, the UK (NICE and SMC), Italy, and Belgium.

Bylvay was evaluated in the ASSERT Phase 3 study for Alagille syndrome (ALGS), which met its primary and key secondary endpoints with highly statistically significant reductions in both pruritus and serum bile acids and was well tolerated. Albireo plans to file supplemental submissions for the use of Bylvay in ALGS with the U.S. Food and Drug Administration and European Medicines Agency immediately. Bylvay is being evaluated in the BOLD Phase 3 study for patients with biliary atresia. The BOLD study remains on track to report topline data in 2024.

For Healthcare Provider General Inquires, please email: France@albireopharma.com

PEDFIC 1 & PEDFIC 2 Studies

The decision by the Committee Economic Health Products is based on data from the PEDFIC 1 and PEDFIC 2 global Phase 3 trials in PFIC. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated with low incidence of drug-related diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients). PEDFIC 2, a long-term, open-label Phase 3 extension study, affirmed Bylvay delivered sustained reductions in serum bile acid, as well as improvements in pruritus assessments, growth and sleep and markers of liver function in patients treated up to 48 weeks in an interim analysis. Across both studies, Bylvay was well tolerated. The most common adverse reactions for Bylvay were diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. There were no serious treatment-related adverse events reported in any clinical study with Bylvay.

About Bylvay (odevixibat)    
The European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA) have granted marketing authorization of Bylvay for the treatment of PFIC in patients aged 6 months or older. Bylvay is also the first drug approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of progressive familial intrahepatic cholestasis (PFIC). Limitation of Use: Bylvay may not be effective in PFIC type 2 patients with ABCB11 variants resulting in non-functional or complete absence of bile salt export pump protein (BSEP-3). A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay can be taken as a capsule for patients that are able to swallow capsules, or opened and sprinkled onto food, which is a factor of key importance for adherence in a pediatric patient population. The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The medicine can only be obtained with a prescription. For more information about using Bylvay, see the package leaflet or contact your doctor or pharmacist. For full prescribing information, visit www.bylvay.com.

In Europe and the U.S., Bylvay has orphan exclusivity for its approved PFIC indications, and orphan designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase 3 study for patients with biliary atresia and the ASSERT open-label trial for ALGS.

Important Safety Information    

• The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.    
• Liver Test Abnormalities: Patients should obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation.    
• Diarrhea: Treat dehydration. Treatment interruption or discontinuation may be required for persistent diarrhea.    
• Fat-Soluble Vitamin (FSV) Deficiency: Patient should obtain baseline vitamin levels and monitor during treatment. Supplement if deficiency is observed. If FSV deficiency persists or worsens despite FSV supplementation, discontinue treatment.    

About Albireo    
Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat pediatric and adult liver diseases. Albireo’s lead product, Bylvay, was approved by the U.S. FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and in Europe for the treatment of PFIC. Bylvay is also being developed to treat other rare pediatric cholestatic liver diseases with a completed Phase 3 trial in Alagille syndrome (ALGS), an ongoing Phase 3 study in biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. The Company has also completed a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing with A2342 for viral and cholestatic liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. For more information on Albireo, please visit www.albireopharma.com.    

Forward-Looking Statements
This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: Albireo’s commercialization plans; the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of Bylvay, A3907, A2342 or any other Albireo product candidate or program; the PEDFIC 2 open-label trial in patients with PFIC; the pivotal trial for Bylvay in biliary atresia (BOLD); the Phase 2 study for A3907 the IND-enabling or clinical studies for A2342; the target indication(s) for development or approval; the timing for initiation or completion of or availability or reporting of results from any clinical trial, including the long-term open-label extension studies for Bylvay in PFIC and Alagille syndrome, the BOLD trial, the Phase 2 study for A3907, and the IND-enabling and clinical studies for A2342; potential regulatory approval and plans for potential commercialization of Bylvay in additional countries; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; or Albireo’s plans, expectations or future operations, financial position, revenues, costs or expenses. Albireo often uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” or the negative of these terms or other similar expressions to identify forward-looking statements. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to: results achieved in Bylvay in the treatment of patients with PFIC may be different than observed in clinical trials, and may vary among patients; potential negative impacts of the COVID-19 pandemic, including on manufacturing, supply, conduct or initiation of clinical trials, or other aspects of our business; whether favorable findings from clinical trials of Bylvay to date, including findings in indications other than PFIC, will be predictive of results from other clinical trials of Bylvay; the timing for initiation or completion of, or for availability of data from, clinical trials of Bylvay, including BOLD and the Phase 2 clinical trial of A3907, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or Europe; delays or other challenges in the recruitment of patients for, or the conduct of, the Company’s clinical trials; and the Company’s critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading “Risk Factors” in Albireo’s most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement except as required by applicable law.

Media Contacts:
Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.com   
Lance Buckley, 917-439-2241, lbuckley@lippetaylor.com  

Investor Contact:
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578