FDA accepts Ipsen’s NDA for ultra-rare genetic disorder drug
pharmafile | June 1, 2021 | News story | Manufacturing and Production | EMA, FDA, FOP, NDA, ipsen
The FDA have accepted Ipsen’s New Drug Application (NDA) for palovarotene, an oral, investigational, selective RARγ agonist for the prevention of heterotopic ossification (new bone formation), as a potential treatment option for patients with the ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP).
The NDA is under Priority Review status and was based primarily upon data from the ongoing Phase III MOVE trial. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualised heterotopic ossification (HO) volume in patients with FOP. Analysis of the primary endpoint data demonstrated a 62% reduction in mean annualised new HO volume in participants treated with palovarotene versus untreated participants from a natural-history study.
Dr Howard Mayer, Executive Vice President and Head of Research and Development at Ipsen, said: “With no approved treatments for this progressive and debilitating disease, there remains a great unmet medical need for the FOP community. This year marks 15 years since the discovery of the mutation in the gene ALK2/ACVR1 which causes FOP, and the palovarotene submission is the first worldwide for a potential treatment in this disease.”
FOP is a progressive disabling genetic disorder with an estimated prevalence of 1.36 per million individuals. It is characterised by new bone formation outside of the normal skeletal system, a process known as heterotopic ossification, which can be preceded by painful soft-tissue swelling or “flare-ups”. 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.
Robert J. Pignolo, Chair of Geriatric Medicine & Gerontology, Mayo Clinic College of Medicine, said: “This FDA filing acceptance marks a significant milestone for Ipsen and those living with this relentless condition of FOP, which we hope will have a significant impact.
“News of this potential new treatment will be welcomed by the FOP community, and we await further updates from the FDA. With ultra-rare conditions by their nature affecting very few people around the world it is so important we continue to make progress and advance the management of diseases like FOP.”
In a statement Ipsen said that in addition to the EMA and Swissmedic MAA validations, the company anticipates additional applications to other regulatory agencies in due course.
Kat Jenkins
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