Albireo Receives FDA Fast Track Designation for A4250

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Albireo Receives FDA Fast Track Designation for A4250

BOSTON, Oct. 17, 2018 (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 Fast Track designation to lead product candidate A4250, an ileal bile acid transporter inhibitor, which is being developed for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare and life-threatening liver disease with no approved pharmacologic treatment option.

In addition to Fast Track designation, A4250 has received several other special regulatory designations for development in PFIC. The FDA has granted A4250 Orphan Drug Designation and Rare Pediatric Disease designation with eligibility to apply for a Priority Review Voucher. The European Medicines Agency (EMA) has granted A4250 Orphan Drug Designation, as well as access to the PRIority MEdicines (PRIME) program, and its Paediatric Committee has agreed to Albireo's A4250 Pediatric Investigation Plan.

“Fast Track designation is a recognition of the serious nature of PFIC and the critical unmet medical need, and it reinforces the potential of A4250 to be the first approved pharmacological treatment,” said Ron Cooper, President and Chief Executive Officer of Albireo. “We are making good progress enrolling patients into PEDFIC-1, our A4250 Phase 3 trial, and Fast Track will be helpful in the submission and approval process.”

The FDA grants Fast Track designation to facilitate development and expedite review of drugs to treat serious conditions and fill an unmet need. The purpose of Fast Track is to get important new drugs to the patient earlier. Fast Track designation gives companies an opportunity for increased communication with the FDA, as well as the option of rolling review, which allows companies to submit completed sections of their new drug application individually. Most company submissions become eligible for accelerated approval and priority review if relevant criteria are met, which reduces the FDA’s target timeline for review and action to 6 months from acceptance for filing. A4250 has been granted Fast Track designation for treatment of pruritus associated with PFIC and could be eligible for all of these benefits.

PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease. Moderate to severe pruritus is a common and problematic clinical presentation of PFIC that can severely diminish quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all patients with PFIC require treatment before age 30.

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 gut. 

The PFIC A4250 program or elements of it have received Fast Track, Rare Pediatric Disease and Orphan Drug designations in the United States. The European Medicines Agency (EMA) has granted A4250 Orphan Drug Designation, as well as access to the PRIority MEdicines (PRIME) program for the treatment of PFIC, and its Paediatric Committee has agreed to Albireo's A4250 Pediatric Investigation Plan.  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

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 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 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            

Media Contact:
Heather Anderson
6 Degrees

Source: Albireo Pharma, Inc.

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Source: Albireo Pharma, Inc.