Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced that the National Institute for Health and Care Excellence (NICE) has issued guidance that recommends Bylvay (odevixibat) for the treatment of all types of progressive familial intrahepatic cholestasis (PFIC) in people aged six months and older. Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), administered as a once-daily treatment. With this final recommendation under the Highly Specialised Technologies (HST) pathway, Bylvay will be funded for use within 90 days in the National Health Service in England, Wales and Northern Ireland.

“It’s incredibly gratifying to see children with PFIC gain access to the first ever non-surgical treatment option for a disease that causes tremendous suffering, in many cases leading to cirrhosis and liver failure within the first 10 years of life,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 & PEDFIC 2 studies. “Odevixibat represents an important advance for patients, allowing us to respond to urgent treatment needs with a drug option that can provide clinically meaningful benefits and the ability to reduce the disease burden for patients and families who otherwise face PEBD surgery or liver transplant.”

“Children living with PFIC have an incurable devastating disease that causes pruritus which was treated with liver transplantation. Until the recommendation from NICE, we’ve had no approved drug options to address this devastating disease,” said Professor Deirdre Kelly, Consultant Paediatric Hepatologist, Liver Unit, Birmingham Women’s and Children’s Hospital and University of Birmingham. “My colleagues and I are delighted that we now have an effective non-surgical treatment option to reduce the burden of this disease on children and families and improve the quality of their lives.”

The final NICE recommendation is aligned to the European Marketing Authorization (EMA) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) approval as the first medicine for the treatment of PFIC, a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. Patients with PFIC have impaired bile flow, or cholestasis, 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 beyond the individuals with PFIC to those caring for them as shown by the 2022 multinational PICTURE study, which revealed PFIC negatively affects caregivers’ quality of life, relationships and career prospects.

"Bylvay is a ground-breaking drug that has the potential to transform the lives of children and young people living with PFIC and the families that care for them who experience terrible suffering and quality of life," said Alison Taylor, patient advocate and outgoing Chief Executive of Children’s Liver Disease Foundation (CLDF). “We are delighted that Bylvay will be available to patients in the UK and commend NICE for their review and recommendation.”

The NICE recommendation is based on data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted in PFIC. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated with low incidence of drug-related diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients). PEDFIC 2, a long-term, open-label Phase 3 extension study, affirmed Bylvay delivered sustained reductions in serum bile acid as well as improvements in pruritus assessments, growth and sleep and markers of liver function in patients treated up to 48 weeks in an interim analysis. Across both studies, Bylvay was well tolerated. The most common adverse reactions for Bylvay were diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. There were no serious treatment-related adverse events reported in any clinical study with Bylvay.

“The positive NICE recommendation was rapidly obtained, within six months of MHRA approval, and it reinforces the significant benefit Bylvay offers to patients. We are pleased that more children will now have access to Bylvay,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Additional pricing and reimbursement discussions for Bylvay are ongoing across Europe and are being accelerated wherever possible. The early response has been encouraging, for example, in France Bylvay has received an SMR ‘Important’ and ASMR III from the HAS Transparency Committee.”

About Bylvay (odevixibat) Bylvay is the first drug approved in the U.S. for the treatment of pruritus in patients 3 months of age and older 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 for the treatment of PFIC in patients aged 6 months or older. Bylvay is available in Germany and the UK and will be available for sale in other European countries following pricing and reimbursement approval. With the final NICE recommendation under the HST pathway, and the Company providing Bylvay according to commercial arrangement, Bylvay will be funded for use within 90 days in the National Health Service in England, Wales and Northern Ireland. Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay acts locally in the small intestine. Bylvay can be taken as a capsule for patients that are able to swallow capsules, or opened and sprinkled onto food, which is a factor of key importance for adherence in a pediatric patient population. The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The medicine can only be obtained with a prescription. 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 Albireo 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 lead 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 with pricing listing in Germany and guidance from the National Institute for Health and Care Excellence (NICE) recommending Bylvay for use in the National Health Service in the England, Wales and Northern Ireland UK. The Company has also completed a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing 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 2019 and 2020 Best Places to Work in Massachusetts. For more information on Albireo, please visit www.albireopharma.com. 

Forward-Looking Statements 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: 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); the pivotal trial for Bylvay in Alagille syndrome (ASSERT); the IND-enabling studies for A2342; the target indication(s) for development or approval; 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, the BOLD and ASSERT trials and the IND-enabling studies for A2342; or the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; 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 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 timing for initiation or completion of, or for availability of data from, clinical trials of Bylvay, including BOLD and ASSERT, 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, the Company’s clinical trials; and the Company’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.

Media Contact:Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.com Lance Buckley, 917-439-2241, lbuckley@lippetaylor.com 

Investor Contact:Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578

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