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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