Ipsen announces positive outcome of FDA Advisory Committee on investigational palovarotene for fibrodysplasia ossificans progressiva

• 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 Sohonos^TM (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:

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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): 31-36
3 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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