Ipsen reports CHMP negative review for use of palovarotene in EU

pharmafile | January 27, 2023 | News story | Research and Development  

Ipsen, a biopharmaceutical company focused on medicines in oncology, rare diseases and neuroscience, have been denied marketing authorisation by the Committee for Medicinal Products for Human Use (CHMP) of the EMA for their drug palovarotene.


Palovarotene is used to treat ultra-rare bone disease fibrodysplasia ossificans progressiva (FOP), a disease which causes permanent and continuous new bone formation in soft and connective tissue – a process known as heterotopic ossification (HO). Palovarotene moderates the interactions between the growth factors, receptors and proteins in the retinoid signalling pathway to reduce new HO.


CHMP’s decision was based on the MOVE phase 3 efficacy and safety trial. MOVE aimed to evaluate the efficacy of palovarotene in reducing new HO volume by using a whole-body computed tomography and comparing that with patients untreated beyond standard of care. It also tried to establish the safety of palovarotene in both adults and paediatric patients.


Ipsen state they are seeking re-evaluation from CHMP, based on data from a different trial. Palovarotene has been given Orphan Drug Designation (OOD) and Priority Review by the FDA and was granted orphan medicine designation by the EMA. It is authorised in Canada, and provisionally in the UAE.


Howard Mayer, executive vice president and head of research and development for Ipsen stated: “We are disappointed with this outcome and will be requesting a re-examination of the CHMP opinion. We continue to work closely with the EMA to address the outstanding concerns that led to the decision today, with the goal of making this investigational medicine available to appropriate patients, living with this debilitating disease, where no other treatment option exists. Ipsen remains committed to bringing new therapeutic options to the FOP community, which has been instrumental in the development of investigational palovarotene through their involvement in clinical trials. We are enormously grateful for their continued support.”


James Spargo

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