Albireo Granted Orphan Drug Designation by FDA For Lead Product Candidate A4250 for Treatment of Biliary Atresia

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Expands A4250 development potential in rare cholestatic liver diseases

Biliary atresia is among the most common rare pediatric liver diseases and is the most common pediatric liver disease for which transplants are done

BOSTON, Jan. 22, 2019 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of biliary atresia, a rare and life-threatening liver disease with no approved pharmacologic treatment option.

A4250 now holds orphan drug designations from both the FDA and EMA for the treatment of progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, biliary atresia and primary biliary cholangitis (PBC). The FDA grants orphan drug designation to novel drugs that seek to treat a rare disease or condition, and provides 7 years of market exclusivity for the product upon regulatory approval.

“We are excited to receive orphan drug designation from the FDA for A4250 for the treatment of biliary atresia,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Building on our recent orphan designation in biliary atresia in the EU, and fast track designation in PFIC and orphan drug designation in Alagille syndrome in the U.S., this recognition continues to reinforce the widespread need to treat rare cholestatic liver diseases and the potential of A4250. We plan to expand the development of A4250 in rare cholestatic liver diseases this year.”

Albireo estimates that biliary atresia is one of the most common rare pediatric liver diseases, affecting between 4.5 and 8.5 in every 100,000 children born worldwide. Biliary atresia is a disease of the liver and bile ducts with symptoms developing about 2-8 weeks after birth. Damaged or absent bile ducts result in bile and bile acids being trapped inside the liver, quickly resulting in damage and scarring of the liver cells (cirrhosis), and even liver failure. With about 80 percent of biliary atresia patients (with or without Kasai hepatoportoenterostomy) needing a liver transplant within the first two decades after birth, biliary atresia is the most common pediatric liver disease for which transplants are done.

About A4250 
A4250 is a first-in-class product candidate being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis (PFIC). A highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), A4250 has minimal systemic exposure and acts locally in the small intestine. 

The PFIC A4250 program or elements of it have received fast track, rare pediatric disease and orphan drug designations in the United States. In addition, the FDA has granted orphan drug designation to A4250 for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The European Medicines Agency (EMA) has granted A4250 orphan designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. Its Paediatric Committee has agreed to Albireo's A4250 Pediatric Investigation Plan for PFIC. EMA also has granted orphan designation to A4250 for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. A4250 is currently being evaluated in a Phase 3 clinical program in patients with PFIC, subtype 1 or 2 (NCT03566238).

About Albireo
Albireo Pharma is a clinical-stage biopharmaceutical company focused through its operating subsidiary on the development of novel bile acid modulators to treat orphan pediatric liver diseases, and other liver and gastrointestinal diseases and disorders. Albireo’s lead product candidate, A4250, is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis. Albireo’s clinical pipeline also includes two Phase 2 product candidates. Albireo’s elobixibat, approved in Japan for the treatment of chronic constipation, is the first ileal bile acid transporter (IBAT) inhibitor approved anywhere in the world. Albireo was spun out from AstraZeneca in 2008. 

Albireo Pharma is located in Boston, Massachusetts, and its key operating subsidiary is located 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: the plans for, or progress, scope, cost, duration or results or timing for availability of results of, development of A4250, including regarding the Phase 3 clinical program for A4250 in patients with PFIC; the target indication(s) for development, the size, design, population, location, conduct, objective, duration or endpoints of any clinical trial, or the timing for initiation or completion of or reporting of results from any clinical trial, including the double-blind Phase 3 PFIC trial for A4250; the size of the PFIC population, the Alagille population, the biliary atresia population or any other disease population for indications that may be targeted by Albireo; the potential benefits or competitive position of A4250; the potential benefits of any regulatory designation, including rare pediatric disease designation, orphan drug designation, orphan designation or fast track designation; Albireo often uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “planned,” “continue,” “guidance,” and 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: whether favorable findings from clinical trials of A4250 to date, including findings in indications other than PFIC, will be predictive of results from the trials comprising the Phase 3 PFIC program or any other clinical trials of A4250; whether either or both of the FDA and EMA will determine that the primary endpoint for their respective evaluations and treatment duration of the double-blind Phase 3 trial in patients with PFIC are sufficient, even if the primary endpoint is met with statistical significance, to support approval of A4250 in the United States or the European Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s) or otherwise; the outcome and interpretation by regulatory authorities of the ongoing third-party study pooling and analyzing of long-term PFIC patient data; the timing for initiation or completion of, or for availability of data from, clinical trials of A4250, including the trials comprising the Phase 3 PFIC program, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or European Union; delays or other challenges in the recruitment of patients for, or the conduct of, the double-blind Phase 3 trial; and Albireo’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.

Investor Contact:
Hans Vitzthum
LifeSci Advisors, LLC.
212-915-2568

Media Contact:
Heather Anderson
6 Degrees
980-938-0260
handerson@6degreespr.com 

Source: Albireo Pharma, Inc. 

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Source: Albireo Pharma, Inc.