The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorisation for Ipsen’s investigational palovarotene as a treatment for fibrodysplasia ossificans progressiva (FOP).
The company says it will be requesting a re-examination of the CHMP opinion, based on scientific data available from the existing palovarotene clinical trial program.
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.
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). The average age of diagnosis is five years old and ultimately FOP shortens the median life expectancy to 56 years.
“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.”
Significant need for a treatment
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.
“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. “We in the FOP community have been waiting a long time for innovations to treat this disabling disease.”