Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare
liver disease company developing novel bile acid modulators, today
announced that the U.S. Food and Drug
Administration (FDA) has accepted the Company’s New Drug
Application (NDA) for odevixibat for the treatment of pruritus in
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 patients with
rare pediatric cholestatic liver diseases, including PFIC, biliary
atresia and Alagille syndrome. The FDA has granted Priority Review
and set a Prescription Drug User Fee Act (PDUFA) goal date of July
20, 2021, supporting the Company’s previous guidance of a planned
launch in the second half of 2021. Odevixibat previously received
Fast Track, Rare Pediatric Disease and Orphan Drug Designations in
the U.S.
“With regulatory submissions completed in record time and now
accepted for review by both the FDA and EMA, odevixibat has the
potential to become the first approved drug treatment for patients
with PFIC,” said Ron Cooper, President and Chief Executive
Officer of Albireo. “Additionally, with no approved treatments,
PFIC presents an exciting commercial opportunity and will pave the
way for expected additional indications in Alagille syndrome and
biliary atresia.”
PFIC is a rare and devastating disorder that causes progressive,
life-threatening liver disease. In many cases, PFIC leads to
cirrhosis and liver failure within the first 10 years of life.
There are no drug therapies currently approved for PFIC, only
surgical options. If approved, odevixibat will provide a
once-daily, oral drug option for children with PFIC.
“As parents of children with PFIC, our focus has long been on
consoling our children who scratch so hard they draw blood and on
conversations about liver transplants,” said Emily Ventura, leader
of PFIC Advocacy and Resource Network (www.pfic.org) and mother to
a PFIC patient. “With this exciting decision from the FDA, we are
one step closer to alleviating our children’s burden and bringing
new hope to what was previously an uncertain future with no drug
treatments available.”
The NDA submission is supported by results from PEDFIC 1 and
PEDFIC 2 Phase 3 studies. PEDFIC 1 was the first and largest,
global, pivotal Phase 3 study conducted in PFIC, which evaluated
the efficacy and tolerability of odevixibat in reducing pruritus
and serum bile acids in a randomized, double-blind,
placebo-controlled trial. In the PEDFIC 1 study, odevixibat met
both the 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 odevixibat treated
patients vs. 5.0% of placebo patients). PEDFIC 2 is a long-term,
open-label Phase 3 extension study.
In Europe, the Company has submitted odevixibat for a Marketing
Authorization Application (MAA) to the EMA seeking approval in
PFIC. Odevixibat is the only IBATi granted accelerated assessment
by the EMA. It has also been granted 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. In addition to PFIC, odevixibat has Orphan Drug
Designations for the treatment of Alagille syndrome, biliary
atresia and primary biliary cholangitis.
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. Odevixibat is also currently
being evaluated in the BOLD Phase 3 trial in patients with biliary
atresia, ASSERT Phase 3 trial in Alagille syndrome and the ongoing
PEDFIC 2 Phase 3 open-label trial in patients with PFIC. The
Company provides an Expanded Access Program for eligible patients
with PFIC in the U.S., Europe, Canada and Australia.
Albireo Commercial DayAlbireo will host a
virtual Commercial Day for investors on February 11, from
11:30-1:00pm EST. The Company will provide an overview of the
long-term corporate strategy, the global market opportunity in rare
pediatric and adult liver disease and the global commercialization
strategy and launch readiness plans for odevixibat in PFIC. Full
event details are available on the Albireo Events &
Presentations page at
ir.albireopharma.com/events-presentations.
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 liver transplants
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 www.pficvoices.com.
About OdevixibatOdevixibat is an
investigational product candidate being developed to treat rare
pediatric cholestatic liver diseases, including PFIC, biliary
atresia and Alagille syndrome (ALGS). A potent, once-daily,
non-systemic 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, the BOLD Phase
3 trial in patients with biliary atresia, and the global Phase 3
ASSERT trial for ALGS.
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 with Phase 3 pivotal trials in
PFIC, Alagille syndrome and biliary atresia. The Company completed
IND-enabling studies for new preclinical candidate A3907 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: 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; the pivotal trial for odevixibat in biliary atresia
(BOLD), and the pivotal trial for odevixibat 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 odevixibat in PFIC, the pivotal
trial for odevixibat in biliary atresia, the pivotal trial for
odevixibat in Alagille syndrome; the potential approval and
commercialization of odevixibat; the potential for odevixibat to
become the first approved drug for PFIC patients; 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: whether the NDA for
odevixibat for the treatment of pruritus in patients with PFIC will
be approved by the FDA and whether the MAA for odevixibat in PFIC
will be approved by the EMA; whether the FDA or EMA will complete
their respective reviews within the target timelines, including the
FDA’s PDUFA goal date; the risk that the NDA will not be approved
despite the FDA’s acceptance of the NDA for review; 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; 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 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., 617-430-7578
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