The European Commission is not granting marketing authorisation for palovarotene in fibrodysplasia ossificans progressiva (FOP), following the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in May 2023 after a re-examination of the dossier.
Already in January 2023, the results of the MOVE trial of palovarotene (Sohonos®) in FOP had not convinced the CHMP. They showed :
- in 107 people with FOP, a 60% reduction in the mean annualised volume of heterotopic ossification compared with what was observed in a natural history study involving 114 participants,
- a 23.5% risk of premature fusion of the conjugation cartilage in young people under the age of 18, a known side-effect of retinoids, which peaks between the ages of 8 and 10, affecting 58.3% of young people in this age group.
Sohonos® has been approved for the treatment of FOP in Canada since January 2022. It is also available in the United Arab Emirates. Lastly, it has just been approved in the United States for eligible patients aged 8 and over in girls and 10 and over in boys, a decision announced in a press release on 16 August 2023.
US FDA approves Ipsen’s Sohonos™ (palovarotene) capsules, the first and only treatment for people with fibrodysplasia ossificans progressiva, Ipsen, press release 16 August 2023.
Ipsen announces European Commission decision on palovarotene for the treatment of FOP, Ipsen, press release 19 July 2023
Ipsen receives CHMP negative opinion, following re-examination of potential first FOP treatment in the E.U., Ipsen, press release 26 May 2023.
Ipsen announces positive outcome of FDA Advisory Committee on investigational palovarotene for fibrodysplasia ossificans progressiva, Ipsen, press release 29 June 2023.
Ipsen receives new FDA PDUFA date for investigational palovarotene for the treatment of people with FOP, Ipsen, press release 16 March 2023.
Ipsen receives Complete Response Letter for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva, Ipsen, press release 23 December 2022
