Rare Daily Staff

Ipsen said following discussions with the U.S. Food and Drug Administration it has withdrawn its New Drug Application for palovarotene, its experimental therapy to treat the rare bone disorder fibrodysplasia ossificans progressiva.

The company said it would resubmit its application once additional data is analyzed.

The decision follows ongoing discussions with the FDA following the acceptance of the New Drug Application for Priority Review, which was announced at the end of May. During the review and ongoing dialogue between Ipsen and the FDA, it was recognized that additional analyses and evaluation of data collected from Ipsen’s phase 3 MOVE and FOP program would be required to progress and complete the review process.

It was agreed between Ipsen and the FDA that it would not be possible to complete this within the current NDA review cycle. As a result, Ipsen has therefore confirmed their withdrawal of the NDA for palovarotene. After recent discussion with FDA, Ipsen plans to resubmit to the FDA upon completion of the additional data analyses.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder with an estimated prevalence of 1.36 per million individuals globally; however, the number of confirmed cases varies by country. It is characterized by new bone formation outside of the normal skeletal system, such as in soft connective tissues, a process known as heterotopic ossification (HO), 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.

Palovarotene is an oral, investigational, selective RARγ agonist for the prevention of heterotropic ossification (new bone formation) as a potential treatment for people living with FOP. The target regulatory action date assigned by the FDA under a Priority Review status for palovarotene was 30 November 2021.

“We remain committed to the FOP community through our clinical programs for Ipsen’s two investigational therapies palovarotene and IPN60130. We recognize the urgency from this community to bring a much-needed treatment option to people living with FOP around the world,” said Howard Mayer, executive vice president and head of research and development for Ipsen. “Unfortunately, as there is no regulatory mechanism to ‘pause’ the current ongoing review process, we have taken the decision to withdraw the NDA for palovarotene to undertake the additional analyses and evaluation needed, with plans to resubmit the data for palovarotene as soon as possible.”

Photo: Howard Mayer, executive vice president and head of research and development for Ipsen

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