Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, today announced the launch of an Expanded Access
Program (EAP) for investigational lead product candidate
odevixibat, a highly potent and selective inhibitor of the ileal
bile acid transporter (IBAT), for patients with progressive
familial intrahepatic cholestasis (PFIC) in the U.S., Canada,
Australia and Europe.
“A tremendous need remains for a pharmacologic treatment option
for PFIC, a life-threatening, ultra-rare, pediatric liver disease
with serious and debilitating symptoms. Through our open-label
PEDFIC 2 trial, and now our Expanded Access Program (EAP), we hope
to increase access to odevixibat for all eligible patients to the
greatest extent possible until odevixibat is commercially
available,” said Ron Cooper, President and Chief Executive
Officer of Albireo. “We are pleased to launch the program in the
U.S., Canada, Australia and Europe, and we are working closely with
local regulators in other countries to offer access and continue to
provide hope for PFIC families worldwide.”
Odevixibat is being evaluated in patients with PFIC in the
PEDFIC 1 Phase 3 trial (NCT03566238), for which topline results are
expected in mid-2020, and the PEDFIC 2 open-label trial
(NCT03659916) evaluating the long-term efficacy and safety of
odevixibat in patients with PFIC. The PEDFIC 2 trial is the
preferred route of access for all patients who qualify, as
determined by their physician. Cohort 1 of the trial allows
patients from the PEDFIC 1 clinical trial to continue treatment
with odevixibat until it is commercially available. Cohort 2
comprises patients who did not participate in the PEDFIC 1 trial
and includes other forms of PFIC not included in PEDFIC 1.
The EAP is available to patients in the U.S., Canada, Australia
and Europe but may be limited depending on drug supply. Timing of
availability in Europe will vary due to country-specific and local
regulations. This program is available for patients with a
clinical diagnosis of PFIC who have no other therapeutic options
and do not qualify for, or have access to, PEDFIC 2 Cohort 2. The
EAP inclusion criteria allow participation by patients with reduced
pruritus and serum bile acid levels compared to our existing
studies. All forms of PFIC (even episodic forms –
e.g., benign recurrent intrahepatic cholestasis, commonly known as
BRIC) are allowed, genetic confirmation of PFIC is not required,
and patients who have had a prior liver transplant may be eligible
for this program, as determined by their physician. For more
information on Albireo’s odevixibat Expanded Access Program, visit
www.albireopharma.com/patients-families/expanded-access-policy.
If you are a physician in the U.S. or Canada who would like to
request EAP access for your patient, contact
Odevixibat@clinigengroup.com. If you are a physician in Australia
or Europe who would like to request EAP access for your patient,
contact medicineaccess@clinigengroup.com.
Our aim is to expand this program to other geographies over
time. If you are a physician, patient
or caregiver of a patient outside of the U.S.,
Canada, Australia, or Europe who may be interested in
odevixibat therapy
access, email medinfo@albireopharma.com with your
interest.
About OdevixibatOdevixibat is a product
candidate being developed to treat rare pediatric cholestatic liver
diseases, including progressive familial intrahepatic cholestasis
(PFIC), biliary atresia and Alagille syndrome. A highly potent and
selective inhibitor of the ileal bile acid transporter (IBAT),
odevixibat has minimal systemic exposure and acts locally in the
small intestine. Odevixibat is being evaluated in a Phase 3
clinical trial, PEDFIC 1, in patients with PFIC (NCT03566238), as
well as a Phase 3 clinical trial, BOLD, in patients with biliary
atresia (NCT04336722). The company anticipates initiating a pivotal
trial of odevixibat for Alagille syndrome by the end of this
year.
The odevixibat PFIC program, or elements of it, have received
fast track, rare pediatric disease and orphan drug designations
in the United States. In addition, the U.S. Food and Drug
Administration (FDA) has granted orphan drug designation
to odevixibat for the treatment of Alagille syndrome, biliary
atresia and primary biliary cholangitis. The European
Medicines Agency (EMA) has granted odevixibat orphan
designation, as well as access to the PRIority MEdicines (PRIME)
scheme for the treatment of PFIC. Its Pediatric Committee has
agreed to Albireo’s odevixibat Pediatric Investigation Plan for
PFIC. EMA also has granted orphan designation to odevixibat for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis.
About AlbireoAlbireo Pharma is a clinical-stage
biopharmaceutical company focused on the development of novel bile
acid modulators to treat orphan pediatric liver diseases, and other
liver and gastrointestinal 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, with a third Phase 3 trial being planned in Alagille
syndrome. Albireo’s clinical pipeline also includes two Phase 2
product candidates. Elobixibat is in Phase 2 development in NAFLD
and NASH. Approved in Japan for the treatment of chronic
constipation, elobixibat is the first ileal bile acid transporter
(IBAT) inhibitor approved anywhere in the world.
Albireo was spun out from AstraZeneca in
2008. Albireo Pharma is located in Boston, Mass.,
and its key operating subsidiary is located 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, elobixibat 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 potential availability of
odevixibat through the EAP, whether the FDA continues to allow
odevixibat to be administered through the EAP, the pivotal trial
for odevixibat in biliary atresia (BOLD), the planned pivotal trial
for odevixibat in Alagille syndrome, and the Phase 2 clinical trial
for elobixibat in NAFLD/NASH; 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 Phase 3 PFIC trial for odevixibat, and the long-term
open-label extension study, the pivotal trial for odevixibat in
biliary atresia, the planned pivotal trial for odevixibat in
Alagille syndrome, or the Phase 2 trial for elobixibat in
NAFLD/NASH; the potential approval and commercialization of
odevixibat; discussions with the FDA or EMA regarding our programs;
the potential benefits or competitive position of odevixibat,
elobixibat, or any other Albireo product candidate or program or
the commercial opportunity in any target indication; the potential
benefits of an orphan drug designation; the period for which
Albireo’s cash resources will be sufficient to fund its operating
requirements (runway); 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 the trials comprising the Phase 3 PFIC
program or any 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, even if the primary endpoint is met with
statistical significance, 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 trials comprising the Phase 3 PFIC program, 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, the double-blind Phase 3 trial or other pivotal 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.
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC, 617-430-7578Media Contact: Claire
LaCagnina, 6 Degrees, 315-765-1462, clacagnina@6degreespr.com
Source: Albireo Pharma, Inc.
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