PARIS, France I June 29, 2022 I Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its resubmitted New Drug Application (NDA) for investigational palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disorder. Ipsen is seeking approval of palovarotene, an oral, selective retinoic-acid receptor gamma (RARγ) agonist for the prevention of heterotopic ossification (HO; new bone formation outside of the normal skeletal system). The FDA has assigned 29 December 2022 as the Prescription Drug User Fee Act goal date, which is on track with anticipated regulatory submission timelines.

“This FDA submission acceptance marks a significant milestone for those living with FOP in the U.S. where no approved treatment options exist today for this progressive and debilitating ultra-rare genetic disorder,” said Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen. “Ipsen is committed to developing life-changing treatments for patients living with rare diseases despite the challenges that exist when there are few, or no precedents to build on when developing a new, innovative medicine for a complex condition. We remain committed to the FOP community, and we hope the Priority Review is one step closer towards getting a much needed treatment to people living with FOP as soon as possible.”

The FDA’s Priority Review designation is assigned to investigational therapies that, if approved, represent a significant improvement in the safety or effectiveness of the treatment compared to the current standard of care.

In January 2022, Health Canada was the first regulatory authority to approve SohonosTM (palovarotene capsules) to reduce the formation of HO in adults and children aged 8 years and above for females and 10 years and above for males with FOP. Sohonos is approved in Canada for the treatment of patients with FOP for both chronic use and for flare-ups in these patient populations. Palovarotene is not currently approved outside of Canada. A Marketing Authorization Application for palovarotene has also been filed in the E.U. in 2021, with responses to questions raised by the European Medicines Agency submitted in June 2022, in line with the ongoing regulatory process. Ipsen anticipates additional applications to other regulatory agencies in due course.

FOP has an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country.1,2 It is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as HO,[3] which can be preceded by painful soft-tissue swelling or ’flare-ups’.2 Flare-up episodes are common and are a substantial contributor to the formation of new HO, however HO can form in the absence of a flare-up. HO, once formed, is irreversible and leads to loss of mobility and shortened life expectancy.3

About palovarotene
Palovarotene is an oral investigational, selective retinoic-acid receptor gamma (RARγ) agonist being developed as a potential treatment for people living with the debilitating ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP). Palovarotene, which received rare pediatric disease and Breakthrough Therapy Designations from the U.S. Food and Drug Administration (FDA) for the potential treatment of FOP, was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. The palovarotene New Drug Application (NDA) was initially accepted by the FDA for Priority Review on May 28, 2021, and Ipsen announced withdrawal of the NDA on August 13, 2021. The resubmission of the palovarotene NDA has been accepted by the FDA for Priority Review in the U.S.

About fibrodysplasia ossificans progressiva (FOP)
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by bone that forms outside the normal skeleton, in muscles, tendons, or soft tissue.3 FOP has an estimated prevalence of 1.36 per million individuals globally; however, the number of confirmed cases varies by country.1,2 The median age at time of FOP diagnosis is five years old.4

About Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With Specialty Care sales of €2.6bn in FY 2021, 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, excluding its Consumer HealthCare business, has around 4,500 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 www.ipsen.com.

1 Lilijesthrom, M & Bogard, B. ‘The global known FOP population’, FOP Drug Development Forum, Boston, MA, 24-25 October 2016.
2 Baujat, G et al. Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases. Orphanet Journal of Rare Diseases. 2017; 12:123.
3 Kaplan, FS, et al. The medical management of fibrodysplasia ossificans progressiva: current treatment considerations. Proc Intl Clin Council FOP. 2019; 1:1-111.
4. Pignolo, RJ et al. Bone. 2020; 134:115274.

SOURCE: Ipsen