- Positive opinion from Committee for Medicinal Products for
Human Use recommending approval of Bylvay®
(odevixibat) based on Phase III ASSERT clinical-trial data in
Alagille syndrome (ALGS) already received in July 2023
- Committee for Orphan Medicinal Products confirms a negative
opinion of its review recommending not to maintain the orphan
designation for Bylvay in ALGS
- Ipsen plans to submit a new Marketing Authorisation Application
for the treatment of ALGS by the end of 2023 under a new brand
name
PARIS, FRANCE, 23 October 2023
– Ipsen (Euronext: IPN: ADR: IPSEY) today announced that the
European Medicines Agency's (EMA) Committee for Orphan Medicinal
Products (COMP) confirmed its negative opinion recommending not to
maintain the orphan designation for Bylvay®
(odevixibat) in Alagille syndrome (ALGS). This is despite a
positive opinion from the Committee for Medicinal Products for
Human Use (CHMP) in July 2023. Orphan designation has a strong
influence on the reimbursement mechanisms and access for patients
to medicines in some countries in the E.U. In order to maintain
Bylvay’s orphan designation in the approved treatment of
progressive familial intrahepatic cholestasis (PFIC), Ipsen is
planning to resubmit to the EMA under a new brand name for the
treatment of ALGS by the end of 2023.
“We stand by our commitment to bring as soon as
possible a much-needed treatment option to patients with Alagille
syndrome and their families in the E.U., as we believe in the
potential benefit this medicine could provide to the Alagille
community,” said Christelle Huguet, Executive Vice President and
Head of Research and Development for Ipsen. “We are disappointed
with the opinion of the COMP as the Orphan Medicinal Product
Regulation aims to stimulate research and development for rare
diseases. The current approach to assessing “significant benefit”
threatens to undermine the aims of this Regulation.”
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 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 received regulatory approval in 2021 in
the U.S. as the first medicine-treatment option for patients aged
three months or 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 also received regulatory approval
in the U.S. for the treatment of cholestatic pruritus in patients
aged 12 months or older with ALGS.
Bylvay received orphan exclusivity for the
treatment of PFIC in the U.S. and the E.U. Bylvay also received
orphan designation for the treatment of ALGS in the U.S. and the
E.U. In a potential future third indication under development, the
rare pediatric cholestatic liver disease biliary atresia, Bylvay
received orphan designation in the U.S. and the E.U. and is in
late-stage development with the Phase III BOLD
trial.
ENDS
ASSERT Phase III clinical trial
data
ASSERT was 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, the Middle East, and Asia Pacific.
The trial enrolled patients aged 0 to 17 years with a genetically
confirmed diagnosis of ALGS. In the primary analysis, the trial 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 the 24 weeks). The trial 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 trial, there were no patient discontinuations and 96% of
patients rolled over into the open-label extension trial. 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 Bylvay®
(odevixibat)
Bylvay is a potent, once-daily, non-systemic
ileal bile acid transport inhibitor 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
or 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 also received
regulatory approval 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.com View 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.
About Alagille syndrome
(ALGS)
ALGS 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 few 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.
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 In March 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
Craig
Marks Vice President, Investor
Relations +44 (0)7584 349 193 |
Nicolas Bogler Investor Relations
Manager +33 6 52 19 98 92 |
Media |
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Jennifer
Moore Senior Director, Global Corporate
Communications +1 (347) 401-8583 Amy
Wolf VP, Head of Corporate Brand Strategy &
Communications +41 79 576 07 23
|
Ioana
Piscociu Senior Manager, Global Media
Relations +33 6 69 09 12 96 |
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