In a landmark development, Ipsen has officially received approval from the U.S. Food and Drug Administration (FDA) for its groundbreaking medication, Sohonos (palovarotene) capsules. This approval marks a significant milestone in the treatment of fibrodysplasia ossificans progressiva (FOP), a rare and debilitating genetic disorder characterized by abnormal bone growth outside the skeletal system.
Sohonos, a retinoid-based medication, has been approved for use in both adult and pediatric patients aged 8 years and older for females and 10 years and older for males with FOP. FOP affects approximately 400 individuals in the U.S. and 900 globally, and until now, there has been no approved medicine to address the underlying cause of the disease.
According to Howard Mayer, Head of Research and Development at Ipsen, “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.”
FOP leads to the formation of new bone outside of the skeleton, severely limiting mobility and causing immense pain. The progression of the disease often leads to the loss of basic functions, such as eating, self-care, and maintaining employment. The majority of individuals with FOP require a wheelchair and full-time caregiver assistance by the age of 30, and the disorder ultimately reduces the median life expectancy to 56 years.
Michelle Davis, Executive Director of the International FOP Association, emphasized the life-altering impact of FOP on patients and their families. She stated, “The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”
The FDA’s approval is based on data from the Phase III MOVE trial, the largest multicenter, open-label trial involving adult and pediatric FOP patients. The trial demonstrated that Sohonos significantly reduces the volume of heterotopic ossification compared to no treatment beyond standard care.
It’s important to note that Sohonos™ comes with specific safety considerations, including warnings about potential embryo-fetal toxicity and premature epiphyseal closure in growing pediatric patients.
This approval not only provides a glimmer of hope for individuals suffering from FOP but also comes with a Rare Pediatric Disease Priority Review Voucher, which can be used for future drug applications not eligible for priority review.
Sohonos’s approval signifies a major leap forward in the treatment landscape for FOP, offering patients and their families a new chance at a better quality of life. Ipsen’s dedication to rare disease research and development is a beacon of hope for those affected by these challenging conditions.