– Listed on the German national price list and all pharmacy software programs –
– Bylvay is approved in U.S., EU and UK as first drug treatment for patients with PFIC –
– Only once-daily drug indicated to treat all forms of PFIC –
BOSTON, Sept. 15, 2021 (GLOBE NEWSWIRE) — Albireo Pharma, Inc. (Nasdaq: ALBO), a rare pediatric liver disease company developing novel bile acid modulators, today announced that Bylvay (odevixibat), the first drug approved in Europe for the treatment of all types of progressive familial intrahepatic cholestasis (PFIC), is now available by prescription to patients in Germany. Bylvay is also approved in the U.S. for the treatment of pruritus in all types of PFIC. A potent, non-systemic ileal bile acid transport inhibitor (IBATi), Bylvay is administered as a once-daily capsule or opened and sprinkled onto soft foods and does not require refrigeration.
“With the availability of Bylvay in Germany, we are now able to offer the first non-surgical treatment option to the PFIC patients who are experiencing difficult developmental and physical symptoms,” said Ekkehard Sturm, M.D., Ph.D., Pediatric Hepatologist and Head of Pediatric Gastroenterology-Hepatology, Liver and Intestinal Transplantation at Children’s Hospital, University of Tüebingen in Germany. “We are hopeful that Bylvay will help patients avoid complications associated with surgery and provide them with much needed relief from their symptoms.”
Bylvay is now available through retail pharmacies. Sales promotion has already begun, with Albireo commercial staff covering the key centers to inform them of the availability of Bylvay for the treatment of PFIC and the patient support services available. Once Bylvay is prescribed, healthcare providers and families will have the option to use Albireo Assist®, which is a customized patient support program built with input from medical experts and patient advocates that aims to support patients and caregivers throughout their treatment journey.
“We are excited to bring the first drug treatment option to PFIC patients in Germany, which has the largest market potential in Europe,” said Pamela Stephenson, Chief Commercial Officer of Albireo. “We have an experienced German team, including, commercial, medical and operations who are on the ground running. Based on our initial conversations with healthcare providers, we know that there is great interest in Bylvay and we are confident in the uptake.”
To support payor decision-making, Albireo has submitted a value dossier to the Joint Federal Committee (G-BA) in Germany with the PEDFIC 1 and PEDFIC 2 Phase 3 data, including long-term data with patients on drug for over two years; natural history information; and a caregiver study to reflect the burden of PFIC. This submission commences the drug benefit assessment process. General national reimbursement will be granted throughout the entire process as well as thereafter.
“The PFIC patients around the world who are experiencing extreme challenges and diminished quality of life underscore our urgency to continue on our path towards global availability,” said Ron Cooper, President and CEO of Albireo.
Albireo has launched Bylvay in the U.S. and is working to commercialize Bylvay in other European countries. Bylvay is currently being evaluated by a number of reimbursement agencies in Europe. Albireo is working closely with all relevant agencies to ensure access for patients in Europe as quickly as possible. Outside the U.S., access to Bylvay is available through our Managed Access Program. For more information on this program, please visit https://www.albireopharma.com/patients-families/patient-access.
Bylvay is currently being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, ASSERT Phase 3 study for Alagille syndrome and in the BOLD Phase 3 study for patients with biliary atresia. The ASSERT and BOLD studies remain on track to report topline data in 2022 and 2024 respectively.
About Bylvay (odevixibat)
Bylvay is the first drug treatment approved in the U.S. for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC). The European Commission (EC) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) have also granted marketing authorization of Bylvay in PFIC and will be available for sale in Europe following pricing and reimbursement approval. A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay acts locally in the small intestine. Bylvay does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. The medicine can only be obtained with a prescription and treatment should be started and supervised by a doctor who has experience in the management of PFIC. For more information about using Bylvay, see the package leaflet or contact your doctor or pharmacist. For full prescribing information, visit www.bylvay.com.
In the U.S. and Europe, Bylvay has orphan exclusivity for its approved PFIC indications, and orphan designations for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase 3 study for patients with biliary atresia and the ASSERT Phase 3 study for Alagille syndrome.
Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases. Albireo’s product, Bylvay, was approved by the U.S. FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and it is also being developed to treat other rare pediatric cholestatic liver diseases with Phase 3 trials in Alagille syndrome and biliary atresia, as well as an Open-label Extension (OLE) study for PFIC. In Europe, Bylvay has been approved for the treatment of PFIC and has been submitted for pricing and reimbursement approval. The Company has also initiated a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies moving ahead with A2342 for viral and cholestatic liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. The Boston Business Journal named Albireo one of the 2020 Best Places to Work in Massachusetts for the second consecutive year. For more information on Albireo, please visit www.albireopharma.com.
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 commercialization plans and expectations for commercializing Bylvay in the U.S. and Europe; estimates of the number of patients impacted by PFIC; expectations about Bylvay’s acceptance by healthcare practitioners to treat PFIC patients;; 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 pivotal trial for Bylvay in biliary atresia (BOLD), and the pivotal trial for Bylvay in Alagille syndrome (ASSERT); the Phase 1 trial for A3907; the target indication(s) for development or approval, the size, design, population, location, conduct, cost, objective, enrollment, duration or endpoints of any clinical trial, or the timing for initiation or completion of or availability or reporting of results from any clinical trial, including the long-term open-label extension study for Bylvay in PFIC, and the BOLD and ASSERT trials; discussions with the FDA or EMA regarding our programs; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; the potential effects of Bylvay of the treatment of PFIC patients and its potential to improve the current standard of care; the potential benefits of an orphan drug designation; the length of time for which Albireo’s cash resources are expected to be sufficient, and the milestones and activities to be funded with those cash resources; 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: there are no guarantees that Bylvay will be commercially successful; we may encounter issues, delays or other challenges in launching or 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; results achieved in Bylvay in the treatment of patients with PFIC once we have launched the product may be different than observed in clinical trials, and may vary among patients; other 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 indications other than PFIC, will be predictive of results from other clinical trials of Bylvay; 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 ASSERT, and the Phase 1 clinical trial of A3907, and the outcomes of such trials; Albireo’s ability to obtain coverage, pricing or reimbursement for approved products in the United States or Europe; delays or other challenges in the recruitment of patients for, or the conduct of, Company’s clinical trials; 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.
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578