Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced U.S. Food
& Drug Administration (FDA) approval of Bylvay (odevixibat),
the first drug approved for the treatment of pruritus in 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. Albireo is launching Bylvay immediately to
accelerate availability for the patients and families impacted by
PFIC.
“Treating children with PFIC can be difficult and frustrating
given the current treatment options. Bylvay gives us a non-surgical
option and will change how we treat PFIC,” said Richard Thompson,
Professor of Molecular Hepatology at King’s College London and
principal investigator of PEDFIC 1 and PEDFIC 2. “With this
approval, my colleagues and I now have the opportunity to revisit
how PFIC patients are being managed and we are hopeful for better
outcomes for these children.”
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
have been available, and without them, most PFIC patients do not
survive past the age of 30. There are an estimated 100,000 patients
with cholestatic liver disease without an approved drug treatment.
Of those patients, there are approximately 15,000 with PFIC
(excluding China and India).
“Until now invasive surgery was the only approved treatment
option. With the approval of Bylvay, parents may find hope in
having a less invasive treatment option available,” said Emily
Ventura, leader of PFIC Advocacy and Resource Network
(www.pfic.org) and mother to a PFIC patient. “As a community, we
experience extreme challenges and diminished quality of life for
children and families with PFIC. Managing the symptoms can be
extremely difficult -- the burden is unimaginable with our kids
suffering physically, emotionally and developmentally.”
The approval of Bylvay was supported by 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 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, reaffirmed Bylvay delivered
sustained reductions in serum bile acids as well as improvements in
pruritus assessments, growth and other markers of liver function in
patients treated up to 48 weeks. 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.
“Bylvay is the first ever approval by the FDA of a drug
developed for a pediatric cholestatic liver disease and provides a
non-surgical treatment for patients living with the burden of
PFIC,” said Ron Cooper, President and CEO of Albireo. “We’re
humbled by the children, families and investigators whose
commitment to our clinical trials will bring hope and treatment
benefit for so many future patients.”
Ready for Bylvay LaunchBylvay is expected to be
packaged and shipped within the coming days. With the immediate
Bylvay launch, Albireo is ready with a focus on access and
reimbursement, sales promotion, and patient support. To support
payor decision-making, Albireo is submitting a compelling value
package with the PEDFIC, gold standard, Phase 3 data, which
includes long-term data with patients on drug for over two years;
natural history information; and a caregiver study to reflect the
burden of PFIC.
Sales promotion will begin immediately. The Albireo and Travere
Therapeutics sales representatives will rapidly cover the 60 key
centers to inform them of the availability of Bylvay for the
treatment of pruritis in PFIC and patient access services.
Once Bylvay is prescribed, HCPs and families will have the
option to use Albireo Assist™, which is a customized patient
support program built with input from patient advocates. The
program features dedicated, US-based regional Care Coordinators
employed by Albireo who will investigate benefits and review
financial assistance options to help ensure optimal patient access.
They will also proactively assist with facilitating dosing changes,
lab work, refill reminders, reauthorization and other
activities.
Bylvay is the first commercially available drug for Albireo in
the United States. With the U.S. approval, the FDA issued
a Rare Pediatric Disease Priority Review Voucher (PRV), which the
Company plans to monetize. The Company had cash and cash
equivalents of $186.3 million as of June 30, 2021 (unaudited) and
anticipates the 2021 operating cash burn will be between $130-$135
million. Excluding any proceeds from the planned sale of the PRV,
the Company believes that its cash and cash equivalents will fund
its operating expenses and capital expenditure requirements into
2023, which should be sufficient to launch Bylvay and expansion
beyond PFIC.
Albireo is also studying the use of Bylvay in other rare
pediatric cholestatic liver 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 the BOLD trial expected in 2024.
Conference CallAlbireo will host a conference
call and webcast tomorrow, July 21, 2021 at 8:30 a.m. ET. To access
the live conference call by phone, dial
877-407-0792 (domestic) or 201-689-8263 (international), and
provide the access code 13720443. Live audio webcast will be
accessible from the Albireo Media & Investors
page ir.albireopharma.com/. To ensure a timely connection to
the webcast, it is recommended that participants register at least
15 minutes prior to the scheduled start time. An archived version
of the webcast will be available for replay on the Events &
Presentations section of the Albireo Media & Investors page for
three months following the event.
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). 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 recommended dosage of Bylvay is 40 mcg/kg once-daily in the
morning with a meal. If there is an inadequate clinical response
after three months, the dosage may be increased in 40 mcg/kg
increments up to 120 mcg/kg once-daily not to exceed a total daily
dose of 6 mg. 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.
The European Commission (EC) has granted marketing authorization
of Bylvay in PFIC and will be available for sale in Europe
following pricing and reimbursement approval. Bylvay has 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 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. Until
now, there were 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 AlbireoAssist™AlbireoAssist is designed
to help patients and families access treatment with Bylvay. The
program features dedicated, regional Care Coordinators to
proactively assist with dosing changes, lab work, refill reminders,
reauthorization and other activities. Care Coordinators will also
help investigate benefits and review financial assistance options.
Families with questions about AlbireoAssist and Albireo’s patient
support programs should contact AlbireoAssist at albireoassist.com
| 855-ALBIREO (855-252-4736) 8am-6pm ET. Families outside the U.S.
should call contact medinfo@albireopharma.com |
857-378-2035.
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 candidate, Bylvay, was approved by the U.S.
FDA as the first for the treatment of pruritus in patients with all
sub-types 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. 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. and Europe; estimates of the
number of patients impacted by PFIC; expectations about Bylvay’s
acceptance by healthcare practitioners to treat PFIC patients;
Albireo’s plans to monetize its Rare Pediatric Disease Priority
Review Voucher; 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; 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 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.
Media Contact:Colleen Alabiso, 857-356-3905,
colleen.alabiso@albireopharma.comLisa Rivero, 617-947-0899,
lisa.rivero@syneoshealth.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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