– Bylvay is approved in the U.S. and EU as only once-daily drug indicated for patients with PFIC
– PFIC is a life-threatening liver disease and the number one cause of pediatric liver transplants in Saudi Arabia –
– King Faisal Research Center was one of the top enrolling sites in the global Phase 3 PEDFIC study –
BOSTON, Aug. 18, 2021 (GLOBE NEWSWIRE) — Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, completed an ex-U.S. commercial distributor partnership with Genpharm Services for the Gulf region. The agreement includes commercialization and distributorship in Saudi Arabia for Bylvay, the first drug approved for progressive familial intrahepatic cholestasis (PFIC). Saudi Arabia has one of the highest rates of PFIC in the world. Bylvay was recently approved in the U.S. for the treatment of pruritus in all subtypes of PFIC and received European Marketing authorization for the treatment of all subtypes of PFIC.
“I am proud that we were the largest regional research site for the PEDFIC Phase 3 studies which generated positive evidence supporting the use of Bylvay in PFIC,” said Mohammed Shagrani, M.D., Consultant of Pediatric Hepatology &Transplant Hepatology, King Faisal Specialist Hospital and Research Centre and principal site investigator of PEDFIC studies. “With the data from the positive PEDFIC studies and regulatory approvals, we can potentially provide PFIC patients with the first drug option with Bylvay in Saudi Arabia.”
PEDFIC 1 was the first and largest, global, pivotal Phase 3 study conducted in PFIC, which evaluated the efficacy and tolerability of Bylvay in reducing pruritus and serum bile acids in a randomized, double-blind, placebo-controlled trial. The global PEDFIC program has a clinical site at King Faisal Specialist Hospital and Research Center located in Riyadh. Dr. Mohammad Shagrani, a renowned clinical expert and key opinion leader in the field of rare hepatic cholestatic diseases, is the principal site investigator. His site actively contributed to the Phase 3 pivotal trial and it remains active in the ongoing Phase 3 open-label extension study of Bylvay.
“Saudi Arabia has an increased prevalence of PFIC and is the leading cause of pediatric liver transplant, making it one of the top 10 commercial market opportunities,” said Ron Cooper, President and Chief Executive Officer of Albireo. “The agreement with Genpharm demonstrates the rapid pace at which Albireo is expanding its footprint into key markets.”
Genpharm will be responsible for regulatory filings, reimbursement submissions, medical and commercial support for Bylvay. The agreement with Genpharm advances Albireo’s global strategy for the commercialization of Bylvay in a key region following European and U.S. regulatory approvals of the use of Bylvay in patients with PFIC based on the PEDFIC study. Albireo is taking a targeted, market-leading approach to commercialization globally, with plans to be the first-to-market for PFIC in most regions where prevalence is high.
“At Genpharm, we strive to bring innovative treatment options to patients, particularly those suffering from rare diseases,” said Karim Smaira, CEO and Co-Founder, Genpharm Services. “Given the lack of drug treatment options, we are proud to work with Albireo to help ensure patients in the GCC have access to Bylvay.”
The partnership agreement with Genpharm follows agreements with Medison Pharma, Ltd. (“Medison”) and GEN İlaç for Bylvay in Israel and Turkey, respectively. Each company is a rare disease market leader in its respective region. Earlier in the year, Albireo also announced a promotional agreement in the U.S. with Travere Therapeutics. Albireo plans to announce additional agreements in other key markets, as part of the Company’s global commercialization strategy.
About Bylvay (odevixibat)
Bylvay is the first drug treatment approved in the U.S. for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC). The European Commission (EC) has 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.
About Genpharm Services
Genpharm Services is a privately held regional pharmaceutical company, focused on Rare Diseases and Specialty therapeutics for the Middle East and North Africa (MENA) region. It was founded in 2012 and has its regional offices in Dubai, UAE. It is led by senior industry executives and a dedicated team of experienced staff. It provides fast-track and early market access, sustainable commercial solutions and strategic advice on medical, regulatory, pricing and launch excellence. Our current strategic partners are Novartis Gene Therapy, Orchard Therapeutics, PTC Therapeutics, Sarepta Therapeutics, Ultragenyx Pharmaceuticals, Biohaven Pharmaceuticals and others. Thanks to our “patient first” approach and our Mantra “Bringing Cures to MENA patients”, we are the first and only company to launch successfully Gene therapy in the region. For more information visit https://www.genpharmservices.
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 subtypes of progressive familial intrahepatic cholestasis (PFIC), and 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; or the potential effects of Bylvay of the treatment of PFIC patients and its potential to improve the current standard of care.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