Fibrodysplasia ossificans progressiva (FOP): a complex name for a very rare genetic disease, which affects just over one person (1.36) in a million, with an average age at diagnosis of only 5 years. Due to this disorder, bone tissue forms outside the skeletal system (heterotopic bone), particularly in the muscles, tendons and soft tissue. Bone formations are irreversible and cause progressive loss of mobility, reducing patients’ life expectancy. To date, there is no cure and this is why the news from overseas is important: the US regulatory agency, the Food and Drug Administration (FDA), will submit the new application for the marketing authorization of a drug for Priority Review experimental.
The experimental drug
The new molecule, palovarotene, was developed for the prevention of heterotopic ossification. From a pharmacological point of view it is an oral selective agonist of the retinoic acid receptor gamma (RARγ). The Priority Review designation is awarded to experimental therapies that, if approved, represent a significant improvement in the safety or efficacy of treatment over the current standard of care. Last January, Health Canada was the first regulatory authority to approve palovarotene capsules in adults and children (ages 8 onwards in females and ages 10 onwards in males) with FOP. The drug is currently only approved in this country, both for chronic use and for flare-ups.
Authorization requests in the US and Europe
“The acceptance of submission by the FDA marks a significant milestone for those living with progressive ossifying fibrodysplasia in the United States, where today there are no approved treatment options for this progressive and debilitating ultra-rare genetic disorder,” he said. said Howard Mayer, Executive Vice President and Head of Research and Development at Ipsen, the pharmaceutical company that submitted the application for approval: a necessary treatment for people living with this pathology “. The application for marketing authorization for palovarotene was also filed in Europe in 2021: the answers to the questions received from the European Medicines Agency were submitted in June 2022, in line with the ongoing regulatory process.