Agreement an essential step for a potential EU marketing authorization application
Completion of PIP would provide an additional two years of market exclusivity
Planned Phase 3 clinical trial a key component of agreed PIP
"Because we are developing A4250 to treat a
pediatric patient population, we are especially pleased that the PDCO, which is responsible for activities on potential medicines for pediatric populations and supporting their development in the
As part of the regulatory process for the registration of new medicines with the EMA, companies are required to agree with PDCO on a PIP that outlines a development program for the investigational product in the pediatric population. The agreed PIP for A4250 includes Albireo's previously announced double-blind, placebo-controlled, Phase 3 clinical trial in patients with PFIC (type 1 or 2), ages six months to 18 years, as well as Albireo's completed Phase 2 study of A4250 in children with cholestatic liver disease. In addition, the PIP includes a small clinical trial in neonates with PFIC, which will be deferred until after completion of the planned Phase 3 trial, and the development of a liquid formulation.
A4250 has received orphan designation in the
In a recently completed Phase 2 clinical trial in children with cholestatic liver disease, A4250 exhibited a favorable overall tolerability profile, and most patients showed both a reduction in serum bile acid levels and an improvement in pruritus across multiple scales after four weeks of treatment with A4250.
A4250 is a first-in-class product candidate in development for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC) and potentially other orphan pediatric cholestatic liver diseases. A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), has minimal systemic exposure and acts locally in the gut.
A4250 has been granted orphan drug designation for PFIC in
Albireo 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 clinical pipeline includes a Phase 3 product candidate, a Phase 2 product candidate and a product candidate for which an application for regulatory approval has been submitted in Japan. Albireo was spun out from AstraZeneca in 2008.
Albireo Pharma is located in Boston,
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: the plans for, or progress or scope of,
development of A4250, including regarding the planned Phase 3 clinical program for A4250 in patients with PFIC or the other activities comprising the pediatric investigation plan for A4250; the timing for initiation or completion of or reporting of results from any clinical trial, including the timing for initiation of the planned Phase 3 PFIC clinical program or pediatric investigation plan for A4250; the competitive position of A4250 or the commercial opportunity in PFIC; 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," 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 and uncertainties, including, but not limited to, risks and uncertainties relating to: whether favorable findings from clinical trials of A4250 to date, including findings in indications other than PFIC, will be predictive of results from future clinical trials of A4250, including the trials comprising the planned Phase 3 PFIC program; whether either or both of the
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