Ipsen receives CHMP negative opinion, following re-examination of potential first FOP treatment in the E.U.
26 Mai 2023 - 2:20PM
Ipsen receives CHMP negative opinion, following re-examination of
potential first FOP treatment in the E.U.
- Palovarotene is the first medicine to be submitted for
regulatory approval for fibrodysplasia ossificans progressiva
(FOP), an ultra-rare disease with an estimated prevalence of 1.36
per million individuals and around 900 people diagnosed
worldwide.1,2
- FOP continuously and permanently causes abnormal bone
formation,3 leading to progressive mobility loss and shortened life
expectancy
- There are currently no disease-modifying treatment options
available in the E.U.
- Regulatory processes are continuing in other countries
including the U.S.
PARIS, FRANCE,
26 May
2023 – Ipsen (Euronext: IPN; ADR:
IPSEY) announced today that the re-examination of palovarotene as a
potential treatment for the ultra-rare bone disease, fibrodysplasia
ossificans progressiva, by the Committee for Medicinal Products for
Human Use (CHMP) of the European Medicines Agency confirms the
negative opinion given in January 2023. Palovarotene is the first
medicine to be submitted for regulatory approval for FOP. The only
treatments currently available in the E.U. are for managing the
symptoms caused by FOP, such as inflammation and chronic pain and
not the underlying disease.
“While everyone at Ipsen who has worked
alongside the FOP community for so many years is extremely
disappointed by this decision, we maintain our steadfast commitment
to bring a new treatment option to the FOP community and our full
attention must now turn to the regulatory processes ongoing in
other countries,” said Howard Mayer, Executive Vice President and
Head of Research and Development for Ipsen. “FOP often starts in
young children and over time, as new bone is formed and accumulates
in joints and other areas of the body, most people lose the ability
to eat and drink on their own, many will need a wheelchair to get
around and life expectancy is shortened. We continue to believe
that our Phase III MOVE trial, the first and largest study to be
conducted in patients with FOP, has shown that palovarotene has the
potential to reduce the new extra-skeletal bone formation caused by
the disease and to slow its progression.”
The CHMP opinion reviewed data from the
palovarotene clinical trial program, including the MOVE study, a
Phase III, multi-center, open-label efficacy and safety trial
conducted in FOP. The primary objectives of MOVE were to evaluate
the efficacy of palovarotene in reducing the volume of new abnormal
bone formation, known as heterotopic ossification (HO), in
pediatric and adult patients with FOP, and to study its safety
profile.4
“Today’s news is a step back for patients with
FOP in the E.U. and for the clinicians managing this chronic and
progressive disease,” said Dr. Genevieve Baujat, Clinical
Geneticist Consultant at Necker-Enfants Malades Hospital, Paris,
France. “Many of my colleagues and I have been waiting for a long
time for a treatment that can help us manage the devastating
disease that is FOP. We got so close, but it seems that in Europe
the wait will continue, while we build on our scientific and
clinical understanding of the disease, which has been informed
through studies like MOVE.”
ENDS
About palovarotenePalovarotene
is an investigational oral medicine that selectively targets the
retinoic-acid receptor gamma (RARγ), which is an important
regulator of skeletal development and ectopic bone in the retinoid
signaling pathway. Palovarotene is designed to mediate the
interactions between the receptors, growth factors and proteins
within the retinoid signaling pathway to reduce new abnormal bone
formation (HO). Palovarotene received Orphan Drug and Breakthrough
Therapy Designations from the U.S. Food and Drug Administration
(FDA) for the potential treatment of FOP and was granted Priority
Review. Palovarotene was also granted orphan medicine designation
by the European Medicines Agency (EMA). Palovarotene is in review
processes with a number of regulatory authorities including the FDA
and the EMA. Palovarotene is currently authorized for use in
appropriate patients only in Canada and provisionally in the U.A.E.
where it is marketed as SohonosTM (palovarotene capsules).6
About the MOVE trialMOVE
(NCT03312634) is a Phase III, multicenter, single-arm, open-label
trial to assess the efficacy and safety of palovarotene. 107 study
participants with FOP received oral palovarotene as a chronic (5mg
once daily) and episodic (20mg once daily for 4 weeks, followed by
10mg for ≥8 weeks for flare-ups and trauma) regimen. The primary
endpoint was annualized change in new HO volume measured by
low-dose whole-body computed tomography.4 Efficacy data from
participants enrolled in MOVE were compared with data from FOP
Natural History Study (NHS) participants untreated beyond standard
of care; individuals ≤65 years of age with clinically diagnosed FOP
and a verified ACVR1R206H pathogenic variant were eligible for
inclusion in the NHS.5
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)7717 801 900 Amy WolfVP, Head
of Corporate Brand Strategy & Communications+41 79 576 07 23
|
Ioana PiscociuSenior
ManagerGlobal Media Relations+33 6 69 09 12 96 |
Ipsen’s
forward-looking
statements
The forward-looking statements, objectives and
targets contained herein are based on Ipsen’s management strategy,
current views and assumptions. Such statements involve known and
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to achieve its financial targets, which were set assuming
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on conditions or facts likely to happen in the future, and not
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in early development phase or clinical trial may end up never being
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face competition from generic medicine that might translate into a
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process involves several stages each of which involves the
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be forced to abandon its efforts with regards to a medicine in
which it has invested significant sums. Therefore, Ipsen cannot be
certain that favorable results obtained during preclinical trials
will be confirmed subsequently during clinical trials, or that the
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and uncertainties include but are not limited to, general industry
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of pharmaceutical industry regulation and healthcare legislation;
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advances, new medicine and patents attained by competitors;
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Ipsen also depends on third parties to develop and market some of
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expressly disclaims any obligation or undertaking to update or
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Marchés Financiers. The risks and uncertainties set out are not
exhaustive and the reader is advised to refer to Ipsen’s latest
Universal Registration Document, available on ipsen.com.
References
- Baujat et. Al. Prevelance of fibrodysplassia ossificans
progressiva (FOP) in France; an estimate based on record linkage of
two national databases. Orphnet J Rare Dis. 2017;12:123
- Lilijesthrom M, et al. Epidemiology of the global
fibrodysplasia ossificans progressiva (FOP) community. J Rare Dis
Res treat. 2020;5(2):31-36
- Kaplan FS et al. The medical management of fibrodysplasia
ossificans progressiva: current treatment considerations. Proc Intl
Clin Council FOP. 2019;1:1-111
- Pignolo RJ, Hsiao E, Al Mukaddam M et al. Reduction of New HO
in the Open-Label, Phase 3 MOVE Trial of Palovarotene for
Fibrodysplasia Ossificans Progressiva (FOP). J Bone Miner Res.
2022.
- Pignolo RJ, Baujat G, Brown M et al. The natural history of
fibrodysplasia ossificans progressiva: A prospective 36-month
study. Gen Med. 2022,ISSN
1098-3600,https://doi.org/10.1016/j.gim.2022.08.013.
- Government of Canada, Notice: Multiple Additions to the
Prescription Drug List (PDL). Viewed 30 November 2022,
<https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/prescription-drug-list/notices-changes/multiple-additions-2022-01-24.html>.
- Ipsen PR_CHMP Palovarotene_26052023
Ipsen (EU:IPN)
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