Albireo Pharma, Inc. (Nasdaq: ALBO), a rare pediatric liver disease
company developing novel bile acid modulators, today announced that
the UK Medicines and Healthcare Products Regulatory Agency (MHRA)
has granted marketing authorization for Bylvay (odevixibat) for the
treatment of all types of progressive familial intrahepatic
cholestasis (PFIC). Bylvay is a potent, non-systemic ileal bile
acid transport inhibitor (IBATi), administered as a once-daily
capsule or opened and sprinkled onto soft foods and which does not
require refrigeration.The MHRA authorization follows the European
Commission (EC) authorization of Bylvay in July 2021.
“The approval of Bylvay gives parents tremendous hope as the
first drug treatment specifically for PFIC to be deemed safe and
effective,” said Alison Taylor, Chief Executive of Children’s Liver
Disease Foundation (CLDF). “The suffering and quality of life for
children with PFIC is terrible, which is why we are so glad that
Bylvay now has broad approval across Europe, and we are optimistic
that it will be made available to patients in the UK.”
The MHRA authorization was 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 markers of liver
function in patients treated up to 48 weeks in an interim analysis.
Across both studies, Bylvay was well tolerated with
diarrhea/frequent stools being the most common treatment-related
gastrointestinal adverse events. There were no serious
treatment-related adverse events reported in any clinical study
with Bylvay.
“The approval of Bylvay in the UK marks an important milestone
to provide global access to the first approved drug for children
with PFIC who desperately need it,” said Ron Cooper, President and
Chief Executive Officer of Albireo. “We are excited that we now
have broad approval across Europe so that we can continue our
global launch efforts to make Bylvay available in over 30 countries
worldwide.”
Albireo has launched Bylvay in the U.S. and is working to
commercialize Bylvay in Europe. Bylvay is currently being evaluated
by NICE under the Highly Specialised Technologies (HST) pathway,
and Albireo is working closely with NICE and NHS England to ensure
access for patients in England and Wales as quickly as possible.
The Company is also actively engaging with the Scottish Medicines
Consortium (SMC). Albireo has developed a compelling value package
with the PEDFIC gold standard Phase 3 data, natural history
information and data from a recent study reflecting the burden of
PFIC on caregivers and families.
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
for the treatment of 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 AlbireoAlbireo 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.
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: Albireo’s commercialization
plans and expectations for commercializing Bylvay in the U.S., the
UK 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, UK 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.
Media Contact: Colleen
Alabiso, 857-356-3905, colleen.alabiso@albireopharma.comLauren
Sneider, 857-300-1737, lauren.sneider@albireopharma.com
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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