Palovarotene

Unassigned

New Medicines

Fibrodysplasia ossificans progressiva

Information

New molecular entity
Ipsen
Ipsen

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Selective retinoic acid receptor gamma agonist; blocks heterotopic ossification
Fibrodysplasia ossificans progressiva (FOP) is an extremely rare congenital disease in which heterotopic ossification (inappropriate bone growth) occurs in voluntary muscles, often in response to local injury. It causes progressive disability and in severe cases eventual near immobility. Estimated UK incidence is 1 per 1.64 million. [3] Estimated world total number is about 2,500 of which about 700 have been confirmed; about 45 in the UK and 285 in the US [4].
Fibrodysplasia ossificans progressiva
Oral