Albireo Presents Data on Greater Efficacy in PFIC with Earlier Bylvay® Treatment at NASPGHAN
– New data presented at the
– Earlier treatment with Bylvay (odevixibat) provided greater efficacy in children with PFIC
– Direct link between level of pruritus relief, liver function, and sleep-related outcomes
– Bylvay improved bile acids, sleep, growth, and quality of life even in patients with low pruritus
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. Lorenzo D’Antiga, Department of Paediatric Hepatology, Gastroenterology, and Transplantation, Azienda Ospedaliera Papa Giovanni XXIII,
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.
New Analyses of the PEDFIC 1 and PEDFIC 2 Trials
The global PEDFIC trials represent the largest studies ever completed in children with PFIC. New pooled data analyses from PEDFIC 1, a randomized, double-blind, placebo-controlled Phase 3 trial that evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids (sBAs) in children with PFIC, and PEDFIC 2, a long-term, open-label Phase 3 extension study, are being shared at NASPGHAN in an oral presentation and two poster presentations. An additional poster presents data showing that Bylvay may be mixed in liquids to give to the youngest patients:
Benefit to Treating Earlier with Bylvay
Oral Presentation: Hepatic Impairment Classifications at Baseline in Responders to Odevixibat Therapy in Children with Progressive Familial Intrahepatic Cholestasis
Lead Author: Dr. Lorenzo D’Antiga, Department of Paediatric Hepatology, Gastroenterology, and Transplantation, Azienda Ospedaliera Papa Giovanni XXIII,
Session Title: Concurrent Session V – Potent P’s in Hepatology: A daily double
Date & Time:
Pooled data analysis showed that treatment with Bylvay is associated with improvements in serum bile acids and pruritus and suggests greater efficacy when treatment with Bylvay occurs earlier in the disease. Significantly more patients with mild hepatic impairment at baseline, according to Child-Pugh scores, had a serum bile acid response than patients with more severe hepatic impairment at baseline. Among patients with mild and moderate hepatic impairment at baseline, 45% and 24% were Bylvay responders, respectively (p value=0.039). No patients had Child-Pugh scores of severe hepatic impairment at baseline. Across baseline hepatic impairment levels, pruritus response rates were comparable and Bylvay was generally well tolerated.
Direct Link Between Sustained Level of Pruritus Reduction and Magnitude of Improvement in Other Disease Parameters
Poster Abstract #301: Outcomes with Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis by Level of Pruritus Reduction: Pooled Analysis from the PEDFIC Trials
Lead Author: Dr.
Session Title: Poster Session II
Date & Time:
An analysis of pooled data from 77 patients treated with Bylvay showed that pruritus reductions were associated with improvements in serum bile acids, hepatic parameters, growth, and disease aspects related to sleep and, in general, patients who had larger-magnitude pruritus reductions had larger-magnitude improvements in other outcomes. The median percentage change from baseline to weeks 70–72 in serum bile acid level was –12% in patients who had pruritus reductions of <1, –54% in patients who had reductions of ≥1 to <2, –94% in patients who had reductions of ≥2 to <3,–96% in patients who had reductions of ≥3 to <4, and –94% in patients who achieved a pruritus score of 0 or 1. Bylvay was generally well tolerated regardless of the level of pruritus reduction.
Evidence of Bylvay Efficacy in Patients with Lower Baseline Pruritus Severity
Poster Abstract #547: Outcomes in Patients with Progressive Familial Intrahepatic Cholestasis Treated with Odevixibat who had Medium or Lower Pruritus Severity at Baseline: Pooled Analysis from the PEDFIC 1 and PEDFIC 2 Studies
Lead Author: Dr.
Session Title: Poster Session III
Date & Time:
A pooled data analysis of 16 patients who had medium or lower pruritus severity at baseline showed that treatment with Bylvay was associated with improvement in serum bile acids, pruritus, hepatic parameters, growth, sleep, and quality of life (QoL), suggesting even patients with lower baseline pruritus severity may benefit from Bylvay treatment. Treatment with Bylvay led to a serum bile acid response in 31% of patients; improvements in mean percentage of days needing help falling asleep, falling from baseline of 41% to 18%; needing soothing falling from 37% to 19%; and sleeping with a caregiver falling from 36% to 16%; but not in percentage of days with scratching associated with bleeding, which was 9% at baseline and 30% at weeks 61-72. Bylvay was generally well tolerated.
Stability of Bylvay Mixed in Liquid for Pediatric Dosing
Poster Abstract #70: Stability of Odevixibat Oral Capsule Contents in Liquids
Lead Author: Dr.
Session Title: Poster Session I
Date & Time:
An in vitro study showed that when Bylvay is mixed in a range of liquids often used to administer medicines to young infants, it is stable and provides children with an adequate dose. As the symptoms of PFIC often begin in infancy, when a child is not yet eating solid food, it is important that parents have an option to mix Bylvay in a liquid to give to their young children.
Albireo will also host a product theater featuring an expert perspective on treating PFIC patients with Bylvay:
Date & Time:
Location: Promenade in front of Exhibit Hall
About Bylvay (odevixibat)
Bylvay is the first drug approved in the
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.
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
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578
Source: Albireo Pharma, Inc.