Ipsen announces positive outcome of FDA Advisory Committee on investigational palovarotene for fibrodysplasia ossificans progressiva
29 Juin 2023 - 2:00AM
- Advisory committee voted 10 for and
4 against that evidence from the Phase III MOVE study show
palovarotene is an effective treatment in patients with the
ultra-rare bone disease fibrodysplasia ossificans progressiva
(FOP)
- Advisory committee further voted 11
for and 3 against on the benefits of palovarotene outweighing the
risks, for the treatment of patients with FOP
- FDA PDUFA action date is anticipated by August 16, 2023
PARIS,
FRANCE, 29 June 2023 – Ipsen
(Euronext: IPN; ADR: IPSEY) announced today that the U.S. Food and
Drug Administration’s (FDA) Endocrinologic and Metabolic Drugs
Advisory Committee (EMDAC) voted in favor of investigational
palovarotene as an effective treatment, with a positive
risk-benefit profile, for people living with the ultra-rare bone
disease, fibrodysplasia ossificans progressiva (FOP). The FDA is
currently reviewing the New Drug Application (NDA) for palovarotene
with a decision anticipated by August 16, 2023. If approved,
palovarotene will be the first treatment in the U.S. for FOP.
“We are pleased with the outcome today and
believe that the vote of the FDA advisory committee conveys the
potential of palovarotene in helping manage the severe impacts of
FOP. For people living with this ultra-rare bone disease, mobility
is severely restricted and they experience a significant loss of
function, all of which completely changes the course of their lives
and can shorten life-expectancy,” said Howard Mayer, Executive Vice
President and Head of Research and Development for Ipsen. “We are
very grateful to the patients and healthcare professionals who have
participated in our clinical trials and to the individuals who
selflessly shared their very personal experiences of living with
FOP during the advisory committee. We will continue to work closely
with the FDA on the next steps.”
The advisory committee voted 10 for and 4
against that evidence from the Phase III MOVE study show
palovarotene is an effective treatment in patients with the
ultra-rare bone disease fibrodysplasia ossificans progressiva
(FOP). The committee further voted 11 for and 3 against on the
benefits of palovarotene outweighing the risks, for the treatment
of patients with FOP.
FOP is an ultra-rare disease that causes
permanent and continuous bone growth in soft and connective tissues
like muscles, tendons and ligaments, also known as heterotopic
ossification or HO.1 FOP impacts the lives of an estimated 400
people in the U.S. and 900 people globally.2,3 As the disease
continuously progresses with flare-up episodes causing rapid bone
growth, FOP severely restricts mobility and function.3 Most people
living with FOP inevitably lose the ability to eat and drink on
their own, can not provide self-care or use the restroom
themselves, and are unable to maintain employment.4 By the age of
30, most people with FOP require a wheelchair and full-time
caregiver assistance.2 Without disease-modifying treatments,
current management is limited to palliative care, and ultimately,
FOP shortens the median life expectancy to 56 years as untimely
death is caused by bone formation around the ribcage leading to
breathing problems and cardiorespiratory failure.2
The advisory committee’s recommendation is based
on its review of the efficacy and safety data package for
palovarotene, that included results from the Phase III MOVE trial,
the first and largest multicenter, open-label trial in adult and
pediatric patients, which demonstrated a clinically meaningful
reduction in new abnormal bone formation (HO) and a
well-characterized safety profile.5
Palovarotene; a potential treatment for
FOP 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 (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 is also under review with a
number of regulatory authorities. Palovarotene is currently
authorized for use in appropriate patients in Canada and United
Arab Emirates where it is marketed as SohonosTM (palovarotene
capsules).6
ENDS
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:
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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Amy
WolfVP, Head of Corporate Brand Strategy &
Communications+41 79 576 07 23 Anna
GibbinsGlobal Head of Franchise Communications,Rare
Disease+44 7717801900 |
Rachel ReiffU.S. Head of
Franchise Communications+1 908 616 1680
Ioana PiscociuSenior
ManagerGlobal Media Relations+33 6 69 09 12 96 |
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1 Kaplan FS, et al. The medical management of fibrodysplasia
ossificans progressiva: current treatment considerations. Proc Intl
Clin Council FOP 1:1-111, 2019.2 Liljesthröm M, Pignolo RJ, Kaplan
FS. Epidemiology of the Global Fibrodysplasia Ossificans
Progressiva (FOP) Community. J Rare Dis Res Treat. (2020) 5(2):
31-363 Pignolo, RJ et al. Bone. 2020; 134:115274.4 Al Mukaddam M,
et al. Val Health 2022;25:S273 (POSA427)5 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. 2022.6 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>.
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