- Positive opinion from Committee for
Medicinal Products for Human Use (CHMP) based on Bylvay Phase III
ASSERT clinical-trial data in Alagille syndrome (ALGS)
- Negative opinion from Committee for
Orphan Medicinal Products (COMP) recommending not to maintain
orphan drug designation for treatment of ALGS
- Ipsen to appeal negative COMP
opinion, which might delay final European Commission decision
- Approval already granted by U.S.
FDA in June 2023 for the treatment of cholestatic pruritus in
patients with ALGS aged 12 months and older
PARIS, FRANCE, 21
July 2023 – Ipsen (Euronext: IPN: ADR:
IPSEY) today announced that the European Medicines Agency's (EMA)
Committee for Medicinal Products for Human Use (CHMP) has issued a
positive opinion recommending the approval of
Bylvay® (odevixibat) for the treatment of
cholestatic pruritus in patients with Alagille syndrome (ALGS) aged
six months or older.
“We are pleased with today’s CHMP positive
opinion which is supported by the data from our Phase III ASSERT
trial,” said Howard Mayer, Executive Vice President and Head of
Research and Development for Ipsen. “These children endure a very
poor quality of life. The severe itching, known as pruritus, caused
by the back-up of bile acids in the liver and bloodstream of
individuals living with Alagille syndrome, sometimes results in
scratching so hard it leads to broken skin. We are committed to
bringing a much-needed additional drug treatment option to Alagille
patients and families in the E.U.”
The Committee for Orphan Medicinal Products
(COMP), a scientific committee of the EMA, has concurrently issued
a negative opinion for the maintenance of Bylvay’s orphan drug
designation in ALGS.This negative COMP opinion prevents the
retention of orphan-drug status in Bylvay’s marketing authorization
in ALGS and might delay a final European Commission decision. Ipsen
plans to submit an appeal in respect of the COMP opinion.
Bylvay, is an orphan medicine already approved
in the E.U. for the treatment of progressive familial intrahepatic
cholestasis (PFIC) in patients aged six months or older. In
November 2022, a variation application to the current market
authorization was submitted. The application sought approval
in the E.U. for a second orphan indication for Bylvay, the
treatment of pruritus in patients with ALGS. In 2012, Bylvay
received orphan designation for the treatment of ALGS in the E.U.,
which supported the development of Bylvay through preclinical and
clinical stages.
The CHMP and COMP reviewed data from the Bylvay
clinical-trial program, including ASSERT, a double-blind,
randomized, placebo-controlled Phase III, multi-center efficacy and
safety trial conducted in ALGS. Positive data from ASSERT presented
at the 2023 European Society for Pediatric Gastroenterology
Hepatology and Nutrition (ESPGHAN) congress, demonstrated that
Bylvay provided highly statistically significant and clinically
meaningful improvements in pruritus, starting as early as one week
after initiation of treatment and were sustained over the 24 weeks
of the trial. More than 90% of patients were pruritus responders (≥
one point change at any time during 24 weeks). The overall
incidence of treatment-emergent adverse events was similar to
placebo. No patients discontinued the trial, and 96% of patients
rolled over into the open-label extension trial.
Bylvay was approved in 2021 in the U.S. as the
first medicine-treatment option for patients three months of age
and older living with cholestatic pruritus due to PFIC, and for the
treatment of PFIC in patients aged six months or older in the E.U.
In June 2023, Bylvay was also approved in the U.S. for the
treatment of cholestatic pruritus in patients from 12 months of age
with ALGS.
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 the E.U. In a
potential future 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
ileal bile acid transport inhibitor (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. In June 2023, Bylvay was also approved in the U.S. for the
treatment of cholestatic pruritus in patients from 12 months of age
with Alagille syndrome.
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.
Approximately 95% of patients with the condition present with
chronic cholestasis, usually within the first three months of life
and as many as 88% also present with severe, intractable pruritus.
The estimated global incidence of ALGS is 3 in 100,000 live births.
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
week 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
On March 3rd, 2023, Ipsen completed the acquisition of Albireo
Pharma Inc, a leading innovator in bile-acid modulators to treat
rare liver conditions, and the marketing authorization holder of
Bylvay.
For further information:
Ipsen
Contacts Investors |
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Craig
MarksVice President, Investor Relations+44 (0)7584 349
193 |
Nicolas BoglerInvestor Relations
Manager+33 6 52 19 98 92 |
Media |
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Jennifer
MooreSenior Director, Global Corporate Communications+1
(347) 401-8583 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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- Ipsen PR_Bylvay CHMP Opinion_21072023
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