Ipsen announces European Commission decision on palovarotene for the treatment of FOP
19 Juli 2023 - 6:42PM
- European Commission determines not
to grant marketing authorization approval for palovarotene for
fibrodysplasia ossificans progressiva (FOP)
- Decision follows negative opinion
by the Committee for Medicinal Products for Human Use, in May
- No currently approved options
available in the E.U. for the treatment of FOP, an ultra-rare
condition, that continuously and permanently causes abnormal bone
formation, leading to progressive mobility loss and shortened life
expectancy
- Ipsen to continue seeking
regulatory approvals in other countries and regions
PARIS, FRANCE,
19 July
2023 – Ipsen (Euronext: IPN; ADR:
IPSEY) announced today that the European Commission has followed
guidance provided by the Committee for Medicinal Products for Human
Use (CHMP) in May this year, and has not granted marketing
authorization for palovarotene, an investigational treatment for
fibrodysplasia ossificans progressiva (FOP). Palovarotene is the
first treatment to be submitted anywhere in the world for
regulatory approval for FOP, an ultra-rare disease with
approximately 900 known cases worldwide.
“We worked tirelessly to bring a greatly needed
treatment option to patients living with FOP in the E.U.,” said
Howard Mayer, Executive Vice President and Head of Research and
Development for Ipsen. “We believe that our clinical data provide
evidence supporting the effect of palovarotene on the reduction of
new, abnormal bone formation, known as heterotopic ossification,
which characterizes the disease. We are therefore disappointed that
the European Commission decided not to approve this treatment for
patients with FOP in Europe. We have learned through this
experience, and we have been motivated by the support of the FOP
community, including those living with the condition and the
doctors and healthcare providers managing their care. This has
encouraged us as we continue to progress with other regulatory
submissions.”
Palovarotene was studied in a comprehensive
clinical program over 15 years. This included MOVE, the first and
largest Phase III clinical trial for FOP, a condition that causes
abnormal bone growth. The average age of diagnosis for FOP is five
years old and the average life expectancy is 56 years old. FOP is a
chronic and progressive condition, where flare-ups occur that can
lead to the development of new, abnormal bone formulation,
accumulating outside of the skeleton in muscles, joints, and other
areas of the body. As a result, most people living with FOP
eventually lose the ability to eat and drink on their own. By the
age of 30 years old, many will need a wheelchair to get around and
full-time care. Life expectancy is shortened, as untimely death can
be caused by bone formation around the ribcage, leading to
breathing problems and cardiorespiratory failure.
“It is devastating to hear that the wait for an
innovative treatment for people living with FOP, and their families
and caregivers will continue, as this negative decision from the
European Commission means that this treatment will not be made
available to patients.” said Dr. Genevieve Baujat, Clinical
Geneticist Consultant at Necker-Enfants Malades Hospital, Paris,
France. “Many of the clinicians that manage patients with this
disease in Europe will have participated in the MOVE clinical trial
and have seen the potential for palovarotene.”
ENDS
About palovarotene
Palovarotene 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. 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. It remains under FDA review, with a
Prescription Drug User Fee Act goal date of 16 August 2023.
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).
About the MOVE trialMOVE
(NCT03312634) was 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. 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.
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
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MarksVice President, Investor Relations+44 (0)7584 349
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Anna
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Disease+44 (0)7717 801 900 Amy WolfVP, Head
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Ioana PiscociuSenior
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- Ipsen PR_EC decision Palovarotene_19072023
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