Ipsen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene as a treatment for the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP).
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In the EU there are currently only symptomatic treatments for FOP, which do not reduce the formation of extra-skeletal bone in patients with the condition.
Ipsen will be requesting a re-examination of the CHMP opinion, based on scientific data available from the existing palovarotene clinical trial program.
FOP causes permanent and continuous new bone formation in soft and connective tissues, like muscles, tendons and ligaments, a process known as heterotopic ossification (HO).1 Once formed, it is irreversible.
The average age of diagnosis is 5 years old2 and ultimately FOP shortens the median life expectancy to 56 years as untimely death is caused by bone formation around the ribcage leading to breathing problems and cardiorespiratory failure.
FOP has an estimated prevalence of 1.36 per million individuals and about 900 people are diagnosed worldwide; however, the number of confirmed cases varies by country.
“We are disappointed with this outcome and will be requesting a re-examination of the CHMP opinion,” said Howard Mayer, Executive Vice President and Head of Research and Development for Ipsen.
“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.”
The CHMP opinion is based on its review of data from MOVE, the first and largest Phase III efficacy and safety trial conducted in FOP.
The primary objectives of MOVE were to evaluate the efficacy of palovarotene in reducing new HO volume, as assessed using whole-body computed tomography, compared with patients untreated beyond standard of care from Ipsen’s global FOP natural history study, and to evaluate the safety of investigational palovarotene in adult and pediatric patients with FOP.
“There is a significant need for a treatment specifically developed to help manage the progression of this disease. The data from MOVE have helped us to understand the potential for treatments that reduce HO progression to be used in the management of FOP.” Said Dr. Genevieve Baujat, Clinical Geneticist Consultant at Necker-Enfants Malades Hospital, Paris, France.
“Currently, the only therapeutic options available to us are for treating symptoms. We in the FOP community have been waiting a long time for innovations to treat this disabling disease.” ■