Ipsen has officially received approval from the U.S. Food and Drug Administration (FDA) for Sohonos™ (palovarotene) capsules. These capsules are now indicated as a retinoid treatment for reducing new heterotopic ossification (HO) volume in adults and pediatric patients aged 8 years and older for females and 10 years and older for males who have fibrodysplasia ossificans progressiva (FOP). This FDA approval marks a significant milestone for the FOP community in the United States, as it represents the availability of an approved medication that has demonstrated effectiveness in reducing the formation of new, abnormal bone growth known as heterotopic ossification (HO).
FOP is a rare disease characterized by continuous progression and flare-up episodes that lead to rapid bone growth, severely restricting mobility and functional abilities. Most individuals with FOP eventually lose the ability to eat, drink, provide self-care, use the restroom independently, and maintain employment. By the age of 30, the majority of people with FOP require a wheelchair and full-time caregiver assistance. Up until now, the management of FOP has been limited to palliative care, and it often reduces the median life expectancy to 56 years. Untimely deaths are often caused by bone formation around the ribcage, which leads to breathing difficulties and cardiorespiratory failure, or falls resulting in fractures or head injuries due to joint ankylosis preventing bracing during a fall.
The FDA's approval decision is based on pivotal efficacy and safety data from the Phase III MOVE trial, which is the first and largest multicenter, open-label trial in adult and pediatric patients with FOP. Sohonos is a groundbreaking oral medication that exhibits specific selectivity for the gamma subtype of retinoic acid receptors. These receptors play a critical role in regulating skeletal development and ectopic bone in the retinoid signaling pathway. Sohonos is engineered to modulate interactions among receptors, growth factors, and proteins within the retinoid signaling pathway, effectively reducing the formation of new abnormal bone in individuals with FOP. The recommended dosing for Sohonos involves a daily dosage of 5 mg (or a weight-based equivalent for pediatric patients under 14 years of age), which can be adjusted or increased as needed for flare-up symptoms. Eligible patients can now receive prescriptions for Sohonos in the United States.
To ensure accessibility to Sohonos for eligible individuals in the U.S., Ipsen Cares has established a patient support program as a valuable resource for people living with FOP. Sohonos has received notable designations from the U.S. Food and Drug Administration (FDA), including Orphan Drug and Breakthrough Therapy status for FOP treatment, and it was granted Priority Review of the New Drug Application (NDA).