Albireo Pharma, Inc.: Italian Medicines Agency Approves Reimbursed Access to Bylvay (odevixibat) For Patients with All PFIC Types

• Positive Agenzia Italiana del Farmaco (AIFA)decision provides reimbursed access to the first medicine available for the treatment of all types of PFIC, a rare and devastating disorder that affects young children and causes progressive, life-threatening liver disease
  
• Bylvay receives "Full Therapeutic Innovation" special designation status by AIFAfor select products with high unmet need, added clinical benefit, robustness of the scientific evidence

BOSTON, Sept. 06, 2022 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced that the Italian Medicines Agency, also known as Agenzia Italiana del Farmaco (AIFA), has enabled reimbursed access to Bylvay (odevixibat) for the treatment of all types of progressive familial intrahepatic cholestasis (PFIC) patients in Italy. 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 causes progressive, liver disease.

"It's extremely important to see the positive decision by AIFA to enable access to Bylvay to treat patients in need. As the first approved medicine for PFIC, I'm relieved to now be able to offer a non-surgical treatment option that provides meaningful clinical benefits that can improve the lives of patients and families," said Dr. Lorenzo D'Antiga, Director, Unit of Hepatology, Gastroenterology and Pediatric Transplantation, Papa Giovanni XXIII Hospital.

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.

"The burden of the disease is devastating for children and families living with PFIC. Severe itching can cause a tremendous impact on patients and disrupt basic daily activities resulting in loss of sleep, poor attention, and lower school performance. Intractable pruritus can, by itself, justify liver transplantation in these patients. We are thrilled to have Bylvay as an innovative therapeutic non-surgical option that could significantly improve disease management and, potentially, modify the natural history of the disease," said Prof. Giuseppe Maggiore, Director of the Division of Hepatology, Gastroenterology, Digestive Endoscopy, and Nutrition, Pediatric Hospital Bambino Gesù.

Bylvay was granted "Full Therapeutic Innovation" status by AIFA, a special designation applied to select drug applications that address a high unmet need, added clinical benefit, and robustness of the scientific evidence. This status provides access to dedicated national funding. For Healthcare Provider General Inquires, please email: [e-mail]

"Bylvay has the potential to transform the lives of those impacted by PFIC, and availability in Italy marks an important milestone for our PFIC community," explained Francesca Lombardozzi, Head of PFIC Italia Network and the mother of a 3-year-old child living with PFIC. "I know firsthand the extreme burden and challenges PFIC patients and caregivers are facing. This is a great first step for the whole community, and we hope others will follow. I'm grateful for the Albireo team's continued efforts to provide access to this critical medication that will make a huge impact on our lives."

"We are grateful to AIFA for this positive decision, recognizing the clinical and economic value of Bylvay," said Thomas Topini, General Manager, Italy at Albireo. "It is because of AIFA's engagement that we were able to accelerate reimbursed access to Bylvay and help PFIC patients and families in Italy living with the devastating effects of this disease."

Bylvay was approved in Europe for the treatment of all types of PFIC in patients aged 6 months or older, based on the PEDFIC 1 study and PEDFIC 2 open-label extension (OLE) study. This availability marks the fourth reimbursement approval for Bylvay in Europe, following launches in Germany, England, Wales & Northern Ireland, and Scotland. Bylvay is currently being evaluated in the ASSERT Phase 3 study for Alagille syndrome (ALGS) and in the BOLD Phase 3 study for patients with biliary atresia. The ASSERT and BOLD studies remain on track to report topline data in the fall of 2022 and 2024, respectively.

PEDFIC 1 & PEDFIC 2 Studies

The decision by the Italian Medicines Agency is based on data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted 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)

Bylvay is 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.

In the U.S. and Europe, 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 Phase 3 study 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.

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 pivotal trial for Bylvay in Alagille syndrome (ASSERT); 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 study for Bylvay in PFIC, the BOLD and ASSERT trials, 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 ASSERT 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, [e-mail]
Lance Buckley, 917-439-2241, [e-mail]

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