FDA Approves First Treatment for Fibrodysplasia Ossificans Progressiva

On August 16, Ipsen announced that the U.S. Food and Drug Administration (FDA) granted approval for their fibrodysplasia ossificans progressiva (FOP) treatment, Sohonos™ (palovarotene). FOP is an ultra-rare disease that is characterized by the development of bones in areas where they aren’t normally found. The approval is based on the positive data from their phase III trial. Sohonos is the first treatment approved by the FDA for FOP, offering much needed treatment to patients suffering from this debilitating disease. 

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Approval Marks a Significant Victory Against Ultra-Rare Disease

The FDA approved the use of Sohonos capsules as a retinoid treatment for FOP. More specifically, it is indicated for the reduction of new heterotopic ossification in adults and pediatric patients. Its use in pediatric patients was approved for those 8 years and older in the case of females and for males 10 years or older. The approval was supported by the efficacy and safety shown by the drug in its phase III MOVE trial. The study showed that palovarotene is effective at reducing heterotopic ossification volume annually. Palovarotene showed the common adverse effects associated with retinoids. These include dry skin, dry lips, and premature growth plate closure in children. 

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease that causes muscles and connective tissue to be replaced with bones. This metamorphosis of tissues causes a progressive locking of joints in patients. Although it only affects about 900 patients globally, it is still an extremely detrimental disease that results in severe restriction of mobility and can lead to loss of the ability to carry out basic functions like eating and drinking. Currently, the treatment for FOP is limited to palliative care. This demonstrates the great unmet need in this area, which will hopefully be addressed to some extent by Sohonos. Ipsen’s Head of Research and Development, Howard Mayer, said, “The FDA approval of Sohonos is a breakthrough for the U.S. FOP community. For the first time doctors have an approved medicine available to them, shown to reduce the formation of new, abnormal bone growth, known as heterotopic ossification (HO), which causes debilitating mobility challenges and has a devastating impact on the lives of people with FOP.”

Breakthrough for FOP Treatment 

Ipsen is a biopharmaceutical company that focuses on the development of treatments in the areas of rare disease, oncology, and neuroscience. Sohonos is the first approved medicine for the treatment of FOP. It is an oral retinoic acid agonist that has selectivity for the gamma subtype of retinoic acid receptors. In patients with FOP, the abnormal bone formation is a result of a mutation in the ACVR1 gene in a pathway that is important in the formation of the skeleton. The drug works by interrupting the signaling pathway responsible for bone formation in FOP. 

Despite this milestone approval, Ipsen did not receive authorization for marketing Sohonos from the European Commission earlier this year. Sohonos has also received Orphan Drug and Breakthrough Therapy Designations and a Rare Pediatric Disease Priority Review (PRV) from the U.S. FDA. This approval is a breakthrough in the treatment of FOP and will have a positive effect on the FOP community as it offers hope for better outcomes. 

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GeneOnline Team