Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced European
Commission (EC) authorization of Bylvay (odevixibat), the first
drug approved for the treatment of all subtypes of progressive
familial intrahepatic cholestasis (PFIC). Bylvay is a potent,
non-systemic ileal bile acid transport inhibitor (IBATi), which
does not require refrigeration and is easily administered as a
once-daily capsule or opened and sprinkled onto soft foods.
“In 1998 one of the genes causing PFIC was discovered in our
laboratory. Bylvay is the first approved pharmacological therapy
available to children after over 20 years of research,” said
Richard Thompson, Professor of Molecular Hepatology at King’s
College London and principal investigator of PEDFIC 1 and PEDFIC 2.
“Until now the standard of care for PFIC patients was limited to
invasive surgeries including liver transplants. As the first
non-surgical treatment option, Bylvay represents a fundamental
shift in the way PFIC is treated.”
PFIC is a rare and devastating disorder affecting young children
that causes progressive, life-threatening liver disease. In many
cases, PFIC leads to cirrhosis and liver failure within the first
10 years of life. The most prominent and problematic ongoing
manifestation of PFIC is pruritus, or intense itching, which often
results in a severely diminished quality of life. Until now, there
have been no approved drugs for PFIC; only surgical options that
include biliary diversion surgery (BDS) and liver transplantation,
and without them most PFIC patients do not survive past the age of
30. Of the estimated 100,000 patients with cholestatic liver
disease worldwide without an approved drug treatment, there are
approximately 15,000 PFIC patients, excluding China and India.
“The suffering and quality of life for children with PFIC is
terrible. The approval of Bylvay gives parents tremendous hope as
the first drug treatment now available,” said Alison Taylor,
Chief Executive of Children’s Liver Disease Foundation
(CLDF). “The decisions we have to make as parents are hard
enough, but for PFIC parents they might have to consider surgery
and liver transplantation for children, while managing an immense
disease burden that affects the entire
family.”
The EC 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 very 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 other 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 Europe marks the first drug ever
approved in multiple countries in the world for a pediatric
cholestatic liver disease and Albireo’s evolution to a
commercial-stage, rare liver disease company,” said Ron Cooper,
President and Chief Executive Officer of Albireo. “We are grateful
to the children, families, investigators, advocates, and employees
whose contributions turned hope into the first ever drug option for
the PFIC community. We are hopeful that Bylvay will soon be
available in the U.S. and other countries around the world to
ensure access for this rare disease population.”
Albireo plans to directly commercialize Bylvay in the European
Union (EU) and is prepared for a global launch with commercial,
market access and medical affairs personnel already on the ground.
This includes Germany, which has the largest EU market potential,
with launch planned for September 2021, following price listing.
Pricing and reimbursement dossiers have been submitted to many
member states to provide access to Bylvay treatment as soon as
possible. 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.
The Company also anticipates an upcoming regulatory decision by
the U.S. FDA on Bylvay for the treatment of pruritus in patients
with PFIC. The FDA has granted a Priority Review for the NDA and
has set an action date of July 20, 2021 under the Prescription
Drug User Fee Act (PDUFA). Albireo is also studying the use of
Bylvay in other rare pediatric cholestatic diseases with the BOLD
Phase 3 clinical trial in patients with biliary atresia and the
ASSERT Phase 3 clinical trial in Alagille syndrome. Topline data
from the ASSERT trial is expected in 2022, and data from the BOLD
trial is expected in 2024.
About Bylvay
(odevixibat)Bylvay is the first drug treatment
approved in the world for the treatment of all subtypes of
progressive familial intrahepatic cholestasis (PFIC). 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.
Bylvay has an Orphan Designation for the treatment of PFIC. The
EMA’s Pediatric Committee has agreed to Albireo’s Bylvay Pediatric
Investigation Plans for PFIC, biliary atresia, and Alagille
syndrome. In addition to PFIC, Bylvay has Orphan Designations for
the treatment of Alagille syndrome, biliary atresia and primary
biliary cholangitis.
Bylvay is available as capsules. The recommended dose is 40
microgram per kilogram body weight. The capsules should be taken
once a day in the morning. They can be taken whole, or they can be
opened and sprinkled on food. If the clinical response is
inadequate after three months, the dose may be increased up to 120
microgram per kilogram body weight. 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 EU product information:
https://albireopharma.com/programs/bylvay.
About PFICProgressive familial intrahepatic
cholestasis (PFIC) is a rare disorder that causes progressive,
life-threatening liver disease. Patients have impaired bile flow,
or cholestasis, caused by genetic mutations. The resulting bile
build-up in liver cells causes liver disease and symptoms. The most
prominent and problematic ongoing manifestation of the disease is
pruritus, or intense itching, which often results in a severely
diminished quality of life. Other symptoms include jaundice, poor
weight gain and slowed growth. In many cases, PFIC leads to
cirrhosis and liver failure within the first 10 years of life, and
nearly all people with PFIC require treatment before age 30. There
are no drugs currently approved for PFIC, only surgical options
that include partial external biliary diversion (PEBD) and liver
transplantation. For information on patient advocacy and disease
education, check out resources from the PFIC Advocacy and Resource
Network at PFIC.org and Children’s Liver Disease Foundation (CLDF)
at childliverdisease.org.
About AlbireoAlbireo Pharma is a rare disease
liver company focused on the development of novel bile acid
modulators to treat rare pediatric and adult liver diseases.
Albireo’s lead product, Bylvay, received European Commission (EC)
authorization as the first drug approved for the treatment of all
subtypes of progressive familial intrahepatic cholestasis (PFIC),
while 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. 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. and
the E.U., including the timing for commercial launch and related
activities; estimates of the number of patients impacted by PFIC;
the potential future approval of Bylvay by the FDA and, if
approved, Albireo’s plans to commercialize Bylvay in the U.S.,
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 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 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; 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; whether the NDA for Bylvay for pruritus in PFIC will be
approved by the FDA; whether the FDA will complete their review
within the target timeline, as a potential result of the impact of
the COVID-19 pandemic or otherwise; whether the FDA will require
additional information, whether we will be able to provide in a
timely manner any additional information that the FDA requests, and
whether such additional information will be satisfactory to the
FDA; 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; whether
the FDA will determine that the primary endpoint for its evaluation
and treatment duration of the double-blind Phase 3 trial in
patients with PFIC are sufficient to support approval of Bylvay in
the U.S., to treat pruritus in PFIC, or otherwise; 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
outcomes of such trials; Albireo’s ability to obtain coverage,
pricing or reimbursement for approved products in the United States
or European Union; 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.com Lisa Rivero, 617-947-0899,
lisa.rivero@syneoshealth.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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