Ipsen RARγ agonist palovarotene enters priority review in the U.S. and Europe

French pharmaceutical company Ipsen (ipsen) recently announced that the U.

If approved, palovarotene will become the world's first drug for the treatment of progressive fibrodysplasia ossificans (FOP).

Skeleton and clinical manifestations of male patients with FOP (picture from literature: DOI:10.

FOP is a very rare autosomal dominant genetic disease, with an estimated prevalence of 1.

Seizures are common and are an important factor in the formation of new HO, but HO can also form without seizures.

Molecular structure of palovarotene (picture source: focusbio.

FOP is caused by ALK2/ACVR1 (activin receptor type IA/activin-like kinase 2, MIM102576) gene mutation.

As a selective RARγ agonist, palovarotene can inhibit BMP signal transduction, thereby preventing heterotopic ossification and delaying the progression of this devastating disease.

Dr.

Mechanism of action of palovarotene (click on the picture to see a larger picture, picture source: mosmedpreparaty.

The palovarotene NDA is mainly based on data from the ongoing MOVE trial (NCT03312634), which is the first global multicenter Phase 3 trial conducted in terms of FOP.

The post-hoc analysis of the primary endpoint of the trial showed that compared with the untreated subjects in the natural history study (n=98, 23318 cubic millimeters), the palovarotene-treated subjects (n=97, 8821 cubic millimeters) had The annual average new HO volume decreased by 62% (nominal weighted linear mixed effects [wLME] model prediction, -11611 cubic millimeters, p=0.

Original source: ipsen Confirms US FDA Accepts New Drug application for Palovarotene as the First Potential Treatment Worldwide for Fibrodysplasia Ossificans Progressiva (FOP)

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