- Approval heralds the second rare
cholestatic liver disease indication for Bylvay in the U.S. after
progressive familial intrahepatic cholestasis related pruritus in
2021
- Immediate U.S. commercial launch
and availability for eligible patients
- ASSERT clinical study demonstrated
efficacy of Bylvay in improvement of pruritus. Also showed
improvement in certain sleep disturbances and reduction in bile
acids – which were secondary endpoints – with a low drug-related
diarrhea rate in patients with Alagille syndrome
- Committee for Medicinal Products
for Human Use opinion expected in Q2 2023 with final European
Medicines Agency decision in second half of 2023
PARIS,
FRANCE, 13 June
2023 – Ipsen (Euronext: IPN: ADR:
IPSEY) today announced that the U.S. Food and Drug Administration
(FDA) has approved Bylvay® (odevixibat) for the
treatment of cholestatic pruritus in patients from 12 months of age
with Alagille syndrome (ALGS). Bylvay is a once-daily, non-systemic
ileal bile acid transport inhibitor (IBATi) that acts locally in
the small intestine and has minimal systemic exposure. Bylvay was
approved as the first drug treatment option for patients living
with cholestatic pruritus due to progressive familial intrahepatic
cholestasis (PFIC) in the U.S., and for the treatment of PFIC in
Europe, in 2021. Bylvay is immediately available via prescription
for eligible ALGS patients.
“Today’s approval of Bylvay in a second
indication allows patients and physicians to access an additional
treatment option that has the potential to improve the management
of pruritus, or intense itch, in this distressing condition that
tends to affect young children,” said Howard Mayer, Executive Vice
President and Head of Research and Development for Ipsen. “We are
proud to have achieved FDA approval for Bylvay as a treatment for
ALGS in the U.S. and we are committed to making it available to
many more eligible patients across the world.”
Positive data from the Phase III ASSERT study,
presented at the 2022 American Association for Study of Liver
Diseases congress, demonstrated that Bylvay provided highly
statistically significant and clinically meaningful sustained
improvements in pruritus, starting early after initiation of
treatment. More than 90% of patients were pruritus responders (≥ 1
point change at any time during 24 weeks). The overall incidence of
treatment-emergent adverse events was similar to placebo. No
patients discontinued the study and 96% of patients rolled over
into the open-label extension study.
“Physicians urgently need more options to treat
patients with Alagille syndrome and this approval from the U.S. FDA
spotlights the robustness of the Phase III ASSERT clinical study
results,” said Nadia Ovchinsky, MD, Chief of the Division of
Gastroenterology and Hepatology, Hassenfeld Children's Hospital at
NYU Langone and ASSERT Principal Investigator. “The ASSERT study
showed that Bylvay reduced pruritus associated with ALGS, which is
so common among this patient population and one of the leading
indications for a liver transplant.”
Roberta Smith, President, Alagille Syndrome
Alliance said: “As an advocate for families impacted by Alagille
syndrome, it is such a blessing to know physicians now have another
drug treatment option for the debilitating pruritus that affects so
many Alagille patients. I know personally the terrible impact of
this rare disease on a child; this approval will help to alleviate
the pruritus burden for more patients.”
Ipsen has also submitted Bylvay to the European
Medicines Agency (EMA), seeking authorization for ALGS, with
Committee for Medicinal Products for Human Use opinion expected in
Q2 2023 and final EMA regulatory decision anticipated in second
half of 2023. Bylvay has received orphan exclusivity for the
treatment of PFIC, and Orphan Drug Designations for the treatment
of ALGS and biliary atresia, in the U.S. and Europe. Bylvay is
already approved in the U.S. for the treatment of
pruritus in patients aged three months and older with all types of
PFIC, and in Europe for the treatment of all types of
PFIC in patients aged six months or older. In a third indication,
the rare pediatric cholestatic liver disease, biliary atresia,
Bylvay is in late-stage development with the Phase III BOLD
trial.
ENDS
About
Bylvay®
(odevixibat)
Bylvay is a potent, once-daily, non-systemic
IBATi that acts locally in the small intestine and has minimal
systemic exposure. It is approved in the U.S. for the treatment of
pruritus in patients three months of age and older with PFIC, where
it has orphan exclusivity. Bylvay was first launched as a treatment
option for patients with PFIC in the U.S. in 2021, where it is
supported by a program designed to assist with access to treatment
and patient support. Bylvay is also approved in the E.U. for the
treatment of PFIC in patients aged six months or older. It has
launched in over nine countries and has secured public
reimbursement across several major markets including Germany,
Italy, the U.K., France and Belgium.
View full U.S. prescribing information here:
Ipsen.comView full E.U. prescribing information here: Bylvay,
INN-odevixibat (europa.eu)
Important Safety
Information
- PFIC: The most common adverse
reactions are diarrhea, liver test abnormalities, vomiting,
abdominal pain, and fat-soluble vitamin deficiency.
- ALGS: The most common adverse
reactions are diarrhea, abdominal pain, hematoma, and weight
decrease.
- Liver Test Abnormalities: Patients
should obtain baseline liver tests and monitor during treatment.
Dose reduction or treatment interruption may be required if
abnormalities occur. For persistent or recurrent liver test
abnormalities, consider treatment discontinuation.
- Diarrhea: Treat dehydration.
Treatment interruption or discontinuation may be required for
persistent diarrhea.
- Fat-Soluble Vitamin (FSV)
Deficiency: Patient should obtain baseline vitamin levels and
monitor during treatment. Supplement if deficiency is observed. If
FSV deficiency persists or worsens despite FSV supplementation,
discontinue treatment.
ALGSALGS is an inherited rare,
genetic disorder that can affect multiple organ systems in the body
including the liver, heart, skeleton, eyes and kidneys. Liver
damage may result from having fewer than normal, narrowed or
malformed bile ducts, which leads to toxic bile acid build-up,
which in turn can cause scarring and progressive liver disease.1
Approximately 95% of patients with the condition present with
chronic cholestasis, usually within the first few months of life
and as many as 88% also present with severe, intractable
pruritus.2,3 The estimated global incidence of ALGS is 3 in 100,000
live births.4 Currently in the U.S., it is estimated that there are
1,300 patients who may be eligible for IBATi treatment.
ASSERT Phase
III Clinical Trial DataASSERT is
a double-blind, randomized, placebo-controlled trial designed to
evaluate the safety and efficacy of 120 µg /kg/day Bylvay for 24
weeks in relieving pruritus in patients with ALGS with 32 sites
across North America, Europe, Middle East,
and Asia Pacific. The trial enrolled patients aged 0 to 17
years of age with a genetically confirmed diagnosis of ALGS. In the
primary analysis, the study met the primary endpoint showing highly
statistically significant improvement in pruritus as measured by
the PRUCISION Observer-Reported Outcome scratching score (0-4 point
scale), from baseline at month 6 (weeks 21 to 24), compared to the
placebo arm (p=0.002). More than 90% of patients were pruritus
responders (≥ 1 point change at any time during 24 weeks). The
study also met the key secondary endpoint showing a highly
statistically significant reduction in serum bile acid
concentration from baseline to the average of weeks 20 and 24
(compared to the placebo arm p=0.001). Statistically significant
improvements in multiple sleep parameters were observed as early as
weeks 1-4 compared to patients on placebo with continued
improvement through week 24. In the study, there were no patient
discontinuations and 96% of patients rolled over into the
open-label extension study. Bylvay had an overall adverse event
incidence similar to placebo and a low incidence of drug-related
diarrhea (11.4% vs. 5.9% placebo).
About Ipsen Ipsen is a
global, mid-sized biopharmaceutical company focused on
transformative medicines in Oncology, Rare Disease and
Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells
medicines in over 100 countries. Alongside its
external-innovation strategy, the Company’s research and
development efforts are focused on its innovative and
differentiated technological platforms located in the heart of
leading biotechnological and life-science hubs: Paris-Saclay,
France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has
around 5,400 colleagues worldwide and is listed in Paris (Euronext:
IPN) and in the U.S. through a Sponsored Level I American
Depositary Receipt program (ADR: IPSEY). For more information,
visit ipsen.com
For further information:
Ipsen Contacts
Investors |
|
Craig
MarksVice President, Investor Relations+44 (0)7584 349 193
|
|
Nicolas BoglerInvestor Relations
Manager+33 6 52 19 98 92 Media |
|
Anna
GibbinsGlobal Head of Franchise Communications,Rare
Disease+44 (0)7717801900 Amy WolfVP, Head
of Corporate Brand Strategy & Communications+41 79 576 07 23
|
Ioana
PiscociuSenior ManagerGlobal
Media Relations+33 6 69 09 12 96 |
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- U.S. Department of Health and Human Services. Alagille
syndrome- about the disease. Genetic and rare diseases information
center.
https://rare-diseases.info.nih.gov/diseases/804/alagille-syndrome
- Singh S P.Euroasian J Hepatogastroenterol.
2018;8(2):140-147
- Feldman A G. Neoreviews 2013;14 (2): e63–e73
- Leonard L. European Journal of Human Genetics. 2014;
22:435
- Ipsen PR_FDA Bylvay_13062023
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