Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease
company developing novel bile acid modulators, today announced the
publication of the PICTURE study in Orphanet Journal of Rare
Disease. A multinational, retrospective, cross-sectional study,
PICTURE evaluated and quantified the impact that progressive
familial intrahepatic cholestasis (PFIC) has on caregivers and
found a significant caregiver-reported burden on health-related
quality of life (HRQoL), impairment of daily activities, reduced
sleep, impact on work productivity, career building challenges and
relationship strain. The study illustrates for the first time that
PFIC levies a substantial burden that extends beyond the
individuals with the disease to those caring for them.
“PFIC is a devastating diagnosis. As a mother of a child with
PFIC, I could not ask for a greater gift than being my daughter’s
advocate. With that said, the PICTURE study reinforces what many of
us intuitively know – the challenges of caregiving are ever present
and often overwhelming and exhausting,” said Emily Ventura, Chief
Executive Director of PFIC Network, co-author of the PICTURE study
and caregiver. “From loss of sleep to stress on finances and
relationships, PICTURE reinforces that we must recognize the burden
this disease puts on families and the importance of providing
resources for PFIC caregivers, while continuing to fund research to
improve treatment and care.”
PFIC is a spectrum of rare, pediatric, genetic diseases of
cholestasis, characterized by inadequate bile secretion, often
requiring liver transplantation, and leading to liver failure and
early death. The rare nature of PFIC has presented challenges to
understanding and quantifying its impact on the daily lives of
patients and their caregivers. The PICTURE study is the first and
largest global study to evaluate and quantify the health-related
quality of life responses from PFIC caregivers. The study showed
that while caregivers report feeling fulfillment from their
caregiving responsibilities, they also reported measurable negative
impacts on many important health-related quality of life measures
and work productivity. Specifically, the study found that:
- PFIC substantially impacted
caregivers’ quality of life
- 82% of caregivers reported a strain
on their relationships.
- 86% of caregivers reported
difficulty sleeping.
- Notably, caregivers in the PICTURE
study reported a median quality of life score of 67.7%. This score
is lower in comparison with quality of life reported in a separate
study of caregivers for individuals with cystic fibrosis, a
similarly rare, genetic, progressive and lifespan-shortening
condition (median 84.7 and 89.2 for mothers and fathers,
respectively).
- On average, PFIC caregivers
reported high impairment in daily activities; more than half also
experienced loss of work productivity
- 50% of caregivers reported an impact
of their child’s PFIC on their career-building efforts, with 73% of
these citing the prevention of either their career progression or
working more hours.
- A third (36%) of caregivers reported
missing an average of 13 workdays in the last three months,
equating to 52 workdays lost in 12 months.
- Of those who were working in paid
employment, 36% stopped work due to caregiving needs, missing an
average of 2.8 years of employment during their career.
“The PICTURE study provides, for the first time, a look at what
it’s really like for caregivers to live with the impact of this
devastating, rare disease and the effect it has every day on
families and adjacent communities,” said Ron Cooper, President and
CEO of Albireo. “At Albireo, we are committed to being the gold
standard partner in the fight against PFIC and the findings from
the PICTURE study reinforce the importance of providing access to
Bylvay, the first approved drug treatment for patients with
PFIC.”
PFIC is a rare and devastating disorder affecting young children
that causes progressive, life-threatening liver disease. Patients
with PFIC have impaired bile flow, or cholestasis, and the
resulting bile build-up in liver cells causes liver disease and
symptoms such as intense itching, poor sleep and diminished quality
of life. Albireo is committed to advancing research in PFIC and
other rare cholestatic diseases. The Company recently received
approval by the U.S. FDA for Bylvay, the first drug for the
treatment of pruritus in all types of PFIC, and in Europe, Bylvay
is approved for the treatment of PFIC. The Company is working with
the community and patient advocacy groups like the PFIC Network and
Children’s Liver Disease Foundation to raise awareness of PFIC and
support families managing the burden of this devastating
disease.
About the PICTURE Study The PICTURE study was a
cross-sectional burden of illness study of physician and
caregiver-reported information for 22 patients with PFIC type 1 or
2 in France, Germany, the United Kingdom and the United States from
September 2020 through to March 2021. Physicians provided clinical
and resource use data of PFIC patients at the time of consultation,
via an electronic Case Report Form (eCRF). Caregivers of PFIC
patients, recruited by the physician as they attended a clinical
appointment with the patient, completed online specific
self-completion questionnaires about the impact of the disease on
their lives. The study was conducted under the guidance of an
Expert Reference Group (ERG), consisting of a representative of
academia as principal investigator, partnering charity and advocacy
representatives as well as experts in the field of liver diseases.
Funding for this study was provided by Albireo Pharma.
About Bylvay (odevixibat) Bylvay is the first
drug approved in the U.S. for the treatment of pruritus in patients
3 months of age and older in all types of progressive familial
intrahepatic cholestasis (PFIC). The European Commission (EC) and
UK Medicines and Healthcare Products Regulatory Agency (MHRA) have
also granted marketing authorization of Bylvay for the treatment of
PFIC in patients aged 6 months or older. Bylvay is available in
Germany and the UK and will be available for sale in other European
countries following pricing and reimbursement approval. A potent,
once-daily, non-systemic ileal bile acid transport inhibitor,
Bylvay acts locally in the small intestine. Bylvay can be taken as
a capsule for patients that are able to swallow capsules, or opened
and sprinkled onto food, which is a factor of key importance for
adherence in a pediatric patient population. The most common
adverse reactions for Bylvay are diarrhea, liver test
abnormalities, vomiting, abdominal pain, and fat-soluble vitamin
deficiency. The medicine can only be obtained with a prescription.
For more information about using Bylvay, see the package leaflet or
contact your doctor or pharmacist. For full prescribing
information, visit https://bylvay.com/.
In the U.S. and Europe, Bylvay has orphan exclusivity for its
approved PFIC indications, and 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 Albireo Albireo Pharma is a rare disease
company focused on the development of novel bile acid modulators to
treat rare pediatric and adult liver diseases. Albireo’s lead
product, Bylvay, was approved by the U.S. FDA as the first drug for
the treatment of pruritus in all types of progressive familial
intrahepatic cholestasis (PFIC), and it is 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 with pricing listing in
Germany and draft guidance from the National Institute for Health
and Care Excellence (NICE), which recommends Bylvay for use in the
National Health Service in the England, Wales and Northern Ireland
UK. The Company has also completed a Phase 1 clinical trial for
A3907 to advance development in adult cholestatic liver disease,
with IND-enabling studies progressing 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 2019 and 2020 Best Places to Work
in Massachusetts. 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 Bylvay, A3907, A2342
or any other Albireo product candidate or program; the pivotal
trial for Bylvay in biliary atresia (BOLD); the pivotal trial for
Bylvay in Alagille syndrome (ASSERT);; the IND-enabling studies for
A2342; the target indication(s) for development or approval; 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, the BOLD and ASSERT
trials and the IND-enabling studies for A2342; or the potential
benefits or competitive position of Bylvay or any other Albireo
product candidate or program or the commercial opportunity in any
target indication; 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:
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
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, the Company’s clinical trials; and
the Company’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.comLance Buckley,
917-439-2241, lbuckley@lippetaylor.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
Von Mär 2024 bis Apr 2024
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
Von Apr 2023 bis Apr 2024