Ipsen receives new FDA PDUFA date for investigational palovarotene for the treatment of people with FOP

• PDUFA date has been set for 16 August 2023 following the New Drug Application resubmission containing additional information on palovarotene clinical trial data, requested in a complete response letter to Ipsen in December 2022

PARIS, FRANCE, 16 March 2023 – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) Prescription Drug User Fee Act (PDUFA) goal date, for the resubmitted New Drug Application (NDA) for investigational palovarotene as a potential treatment for fibrodysplasia ossificans progressiva (FOP), is 16 August 2023. Additional information on palovarotene clinical trial data, requested in a complete response letter to Ipsen in December 2022, will be reviewed as part of this resubmission process.

If approved, palovarotene, which has received FDA Breakthrough Therapy and Orphan Drug designations, would be the first treatment for an estimated 400 people in the U.S. living with FOP, an ultra-rare, progressive, life-limiting bone disease.^1,2

Data submitted to the FDA include additional analyses from across the palovarotene clinical trial program, including the pivotal MOVE study,³ the first Phase III study carried out in people with FOP.

Furthermore, Ipsen has requested a re-examination of the European Medicines Agency’s January 2023 Committee for Medicinal Products for Human Use (CHMP) opinion on palovarotene.

Palovarotene is authorized for use in appropriate patients in Canada where it is marketed as Sohonos^TM (palovarotene capsules). It also has conditional approval in United Arab Emirates. Investigational palovarotene is under review with several regulatory authorities.

ENDS

About FOP

FOP impacts the lives of an estimated 900 people globally.^1,2 The disease continuously progresses with flare-up episodes causing rapid bone growth. Most people living with FOP inevitably lose the ability to eat and drink on their own, cannot provide self-care or use the restroom themselves, and are unable to maintain employment.⁴ By the age of 30 years old, the majority of people with FOP require a wheelchair and full-time caregiver assistance.¹ 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.⁴

About the MOVE trial

MOVE (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 (Heterotopic Ossification) 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.

Results from the post-hoc interim three analysis demonstrated a clinically meaningful 60 percent reduction in new abnormal bone formation HO volume in participants treated with palovarotene, compared to patients on standard of care. Palovarotene also demonstrated a well-characterized safety profile consistent with other therapies in the systemic retinoids class.³

These data were published in the Journal of Bone and Mineral Research in December 2022

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 5,000 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

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1. 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
2. Pignolo, RJ et al. Bone. 2020; 134:115274.
3. Pignolo, RJ et al. J Bone Miner Res. https://doi.org/10.1002/jbmr.4762
4. Al Mukaddam M, et al. Val Health 2022;25:S273 (POSA427)