Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver
disease company developing novel bile acid modulators, today
announced it has submitted a New Drug Application (NDA) to the U.S.
Food and Drug Administration (FDA) and a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) seeking
approval of odevixibat for the treatment of patients with
progressive familial intrahepatic cholestasis (PFIC). Odevixibat is
a potent, once-daily, non-systemic ileal bile acid transport
inhibitor (IBATi) being developed to treat rare pediatric
cholestatic liver diseases, including PFIC, biliary atresia and
Alagille syndrome. The EMA has validated the odevixibat MAA on the
accelerated assessment timeline, which begins the formal review
process. With FDA and EMA regulatory submissions complete,
odevixibat has the potential to become the first approved drug
treatment for patients with PFIC in the U.S and Europe.
Odevixibat has previously received Fast Track, Rare Pediatric
Disease and Orphan Drug Designations in the U.S. In addition to
PFIC, odevixibat has Orphan Drug Designations for the treatment of
Alagille Syndrome, biliary atresia and primary biliary cholangitis.
The EMA has granted odevixibat accelerated assessment, Orphan
Designation, as well as access to the PRIority MEdicines (PRIME)
scheme for the treatment of PFIC. The EMA’s Pediatric Committee has
agreed to Albireo’s odevixibat Pediatric Investigation Plans for
PFIC and biliary atresia. With U.S. and EU regulatory submissions
for odevixibat in PFIC completed, the Company anticipates potential
regulatory approvals, issuance of a rare pediatric disease priority
review voucher and launch in the second half of 2021.
“We have completed both the U.S. and EU regulatory submissions
in record time, which speaks to the Albireo team’s commitment to
providing children with different forms of PFIC a treatment option
as quickly as possible,” said Ron Cooper, President and Chief
Executive Officer of Albireo. “With randomized, placebo-controlled
PEDFIC data, Orphan Designations in both the U.S. and EU,
accelerated assessment and access to the PRIME scheme in the EU and
Fast Track designation in the U.S., we’re on track for potential
approval, launch and broad global access to odevixibat for PFIC
patients in the second half of 2021.”
Phase 3 data was recently presented at the AASLD that showed a
durable response to odevixibat in patients with PFIC. Full results
from PEDFIC 1, the first and largest, global, Phase 3 study ever
conducted in PFIC, confirm both U.S. and EU primary endpoints were
met in the randomized, double-blind, placebo-controlled trial.
Additionally, long-term data from PEDFIC 2, an open-label Phase 3
extension study, demonstrate continued and durable reductions in
sBAs, improvements in pruritus assessments and encouraging markers
of liver and growth function in patients treated up to 48 weeks.
Across both studies, odevixibat was generally well tolerated, and
treatment-emergent adverse events (TEAEs) were mostly mild or
moderate. Collectively, these studies reaffirm odevixibat’s
potential to be the first drug treatment approved for patients
living with PFIC, a devastating disease which is currently treated
with surgical options including liver transplantation.
“With strong data from the first and largest global Phase 3
study ever conducted in PFIC, we have a comprehensive database that
has the potential to influence the way PFIC is treated, the use of
odevixibat and how reimbursement will be achieved,” added Cooper.
“We are grateful to the patients, families and investigators for
their involvement in our mission to bring hope to families and
reduce disease burden. Beyond PFIC, we are poised to initiate our
Phase 3 trial in Alagille syndrome by end of year, expanding our
pivotal programs across three rare liver diseases.”
Odevixibat is also currently being evaluated in the ongoing
PEDFIC 2 Phase 3 open-label trial in patients with PFIC, and the
BOLD Phase 3 trial in patients with biliary atresia. The Phase 3
trial of odevixibat in Alagille syndrome will be the third pivotal
trial of odevixibat. The Company also provides an Expanded Access
Program for eligible patients with PFIC in the U.S., Canada,
Australia and Europe.
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. Additional information on PFIC is available
at https://www.pficvoices.com.
About Biliary
AtresiaBiliary atresia is a rare
pediatric liver disease with symptoms typically developing about
two to eight weeks after birth. Damaged or absent bile ducts
outside the liver result in bile and bile acids being trapped
inside the liver, quickly resulting in cirrhosis, and even liver
failure. Children have clay-colored or no color in their stools,
jaundice among other things and a few patients are pruritic.
Biliary atresia is the most common pediatric cholestatic liver
disease and is the leading cause of liver transplants among
children as there are no approved drug treatments.
About Alagille SyndromeAlagille Syndrome (ALGS)
is a rare multisystem genetic disorder that can affect the liver,
heart, skeleton, eyes, central nervous system, kidneys, and facial
features. Liver damage is caused by a paucity of bile ducts
preventing bile flow from the liver to the small intestine.
Approximately 95 percent of patients with ALGS present with chronic
cholestasis, usually within the first three months of life, and up
to 88 percent also present with severe, intractable pruritus.
Currently, there are no approved drug treatments.
About OdevixibatOdevixibat is an
investigational product candidate being developed to treat rare
pediatric cholestatic liver diseases, including progressive
familial intrahepatic cholestasis (PFIC), biliary atresia and
Alagille syndrome. A potent, once-daily, non-systemic ileal bile
acid transport inhibitor (IBATi), odevixibat acts locally in the
small intestine. Odevixibat 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. Odevixibat is
currently being evaluated in the ongoing PEDFIC 2 open-label trial
(NCT03659916) and the BOLD Phase 3 trial in patients with biliary
atresia (NCT04336722). Initiation of a pivotal Phase 3 trial of
odevixibat for Alagille syndrome is also anticipated by the end of
2020.
About AlbireoAlbireo Pharma is a clinical-stage
biopharmaceutical company focused on the development of novel bile
acid modulators to treat rare pediatric and adult liver diseases,
and other adult liver diseases and disorders. Albireo’s lead
product candidate, odevixibat, is being developed to treat rare
pediatric cholestatic liver diseases and is in Phase 3 development
in progressive familial intrahepatic cholestasis (PFIC) and biliary
atresia, and the first site initiation for the Phase 3 trial in
Alagille syndrome is planned for this month. The Company expects to
complete IND-enabling studies for new preclinical candidate A3907
this year and plans to advance development in adult 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:
the plans for, or progress, scope, cost, initiation, duration,
enrollment, results or timing for availability of results of,
development of odevixibat or any other Albireo product candidate or
program, including regarding expectations regarding the impact of
COVID-19 on our business and our ability to adapt our approach as
appropriate; the Phase 3 clinical program for odevixibat in
patients with PFIC, the pivotal trial for odevixibat in biliary
atresia (BOLD), and the planned pivotal trial for odevixibat in
Alagille syndrome; 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 odevixibat in PFIC, the
pivotal trial for odevixibat in biliary atresia, the planned
pivotal trial for odevixibat in Alagille syndrome; the potential
approval and commercialization of odevixibat; discussions with the
FDA or EMA regarding our programs; the potential benefits or
competitive position of odevixibat or any other Albireo product
candidate or program or the commercial opportunity in any target
indication; the potential effects of odevixibat 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
potential issuance of a rare pediatric disease priority review
voucher; 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,” and 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: 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 odevixibat to
date, including findings in indications other than PFIC, will be
predictive of results from other clinical trials of odevixibat;
whether either or both of the FDA and EMA will determine
that the primary endpoint for their respective evaluations and
treatment duration of the double-blind Phase 3 trial in patients
with PFIC are sufficient to support approval of odevixibat
in the United States or the European Union, to treat
PFIC, a symptom of PFIC, a specific PFIC subtype(s) 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 odevixibat, including
the pivotal program in biliary atresia or the planned pivotal
program in Alagille syndrome, 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.comLisa Rivero,
617-947-0899, lisa.rivero@syneoshealth.com
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC., 857-272-6177
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
From Mar 2024 to Apr 2024
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
From Apr 2023 to Apr 2024