A new analysis of pooled data from the landmark Phase 3 PEDFIC 1 and PEDFIC 2 trials demonstrates that Bylvay (odevixibat) reduces serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) who have varying levels of baseline hepatic impairment. Importantly, the analysis shows that significantly more patients with mild hepatic impairment at baseline had a serum bile acid response, suggesting children may benefit from earlier treatment with Bylvay.
Additional data analyses presented at the meeting show that Bylvay reduced pruritus in patients regardless of their baseline pruritus scores. Further, pruritus reductions were associated with improvements in serum bile acids, hepatic parameters, growth, and disease aspects related to sleep, with greater reductions in pruritus associated, in general, with greater improvements across these additional measures.
'These new data provide important evidence that treating children with Bylvay earlier in their disease course could result in greater efficacy,' said
'If a physician is trying to determine when to start treatment with Bylvay, this new data suggest the earlier the better,' said Dr.
PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease. Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms, such as intense itching, poor sleep, delayed growth, and diminished quality of life. The harmful impacts of the disease extend to parents and caregivers, as the 2022 multinational PICTURE study revealed that PFIC negatively affects caregivers' quality of life, relationships, and career prospects.
About Bylvay (odevixibat)
Bylvay is the first drug approved in the
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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 expected cash runway; 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 target indication(s) for development or approval; the timing for anticipated regulatory filings; discussions with the FDA or EMA regarding our programs; potential regulatory approval and plans for potential commercialization of Bylvay in biliary atresia or ALGS or Albireo's other product candidates; the impact of the Expanded Access Program; 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: whether the regulatory filings to be made for Bylvay in patients with ALGS will be made on the timelines we expect and be approved by the FDA and EMA; whether the FDA and EMA will complete their respective reviews within target timelines, once determined; whether the FDA and EMA will require additional information, whether we will be able to provide in a timely manner any additional information that the FDA and EMA request, and whether such additional information will be satisfactory to the FDA and EMA; there are no guarantees that Bylvay will be commercially successful; we may encounter issues, delays or other challenges in commercializing Bylvay; whether Bylvay receives adequate reimbursement from third-party payors; the degree to which Bylvay receives acceptance from patients and physicians for its approved indication; challenges associated with execution of our sales activities, which in each case could limit the potential of our product; challenges associated with supply and distribution activities, which in each case could limit our sales and the availability of our product; results achieved in Bylvay in the treatment of patients with PFIC or other approved indications 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 PFIC, ALGS and other indications, will be predictive of results from other clinical trials of Bylvay; there is no guarantee that Bylvay will be approved in jurisdictions or for indications (such as biliary atresia or ALGS) beyond the jurisdictions in which or indications for which Bylvay is currently approved; there is no guarantee that our other product candidates will be approved; estimates of the addressable patient population for target indications may prove to be incorrect; 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 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
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