Ipsen SA, a French drugmaker, has gained clearance from the US Food and Drug Administration (FDA) for its drug Sohonos, which treats fibrodysplasia ossificans progressiva (FOP), a rare condition that causes abnormal bone growth. This approval marks the first-ever treatment for FOP in the US. However, the drug’s high price tag of approximately $624,000 per year has raised concerns. Ipsen’s Sohonos was rejected earlier this year by the European Medicines Agency (EMA) due to concerns about its benefits and risks. Despite this setback, patient groups in the US advocated for the drug’s approval, highlighting the lack of alternative treatments available. Ipsen plans to offer financial and insurance assistance programs to help patients with the cost of the medication.
Ipsen’s Drug for Rare Bone Disorder Approved by FDA, Costing $624,000 a Year in the US
French drugmaker Ipsen SA has received clearance from the US Food and Drug Administration (FDA) for its drug Sohonos (palovarotene), making it the first medicine approved to treat fibrodysplasia ossificans progressiva (FOP), a rare disorder characterized by abnormal bone growth. The US approval comes after the drug was rejected by the European Medicines Agency earlier this year due to concerns about its benefits and risks. However, patient groups in the US advocated for the approval of the Ipsen drug since there are no other treatments available for FOP.
According to Ipsen, the cost of Sohonos will vary based on an individual’s weight and disease stage, but the average estimated annual cost based on daily dosing of 5 mg is approximately $624,000. The price does not include out-of-pocket costs, but the company plans to provide financial and insurance assistance programs for patients. Ipsen’s shares fell 0.6% in Paris following the news, although the company’s stock has seen a 20% increase this year.
Sohonos is the first drug shown to reduce the formation of new abnormal bone growth associated with FOP. It works by binding with a protein involved in bone formation, inhibiting growth. The FDA initially requested additional follow-up data from Ipsen after an initial review last year. However, a panel of advisers to the agency voted 11-3 in favor of the drug in June, stating that its benefits outweigh its risks, considering the rarity of the disease.
FOP affects approximately 800 people worldwide, with 400 of those patients residing in the US, according to Ipsen. The disease progressively limits mobility and function, eventually leading to the need for full-time caregiver assistance. Bone formation around the ribcage can also cause breathing difficulties and cardiorespiratory failure. Ipsen will continue to monitor the real-world safety and effectiveness of Sohonos and is also studying a second experimental FOP treatment called fidrisertib in human trials.
In conclusion, Ipsen’s drug Sohonos has been approved by the FDA as the first treatment for fibrodysplasia ossificans progressiva (FOP), a rare bone disorder. The drug’s cost in the US is estimated to be around $624,000 per year, although this can vary based on individual factors. Sohonos works by reducing the formation of abnormal bone growth and has been shown to have benefits that outweigh its risks. FOP affects a small number of people worldwide and severely impacts mobility and function. Ipsen will continue to monitor the drug’s safety and effectiveness while exploring other potential treatments for FOP.
Takeaways:
- Ipsen’s drug Sohonos has received FDA approval for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare bone disorder.
- The drug will cost approximately $624,000 per year in the US, with variations based on individual factors.
- Sohonos is the first drug shown to reduce the formation of abnormal bone growth in FOP patients.
- FOP severely restricts mobility and function, leading to the need for full-time caregiver assistance.
- Ipsen will continue to monitor the drug’s safety and effectiveness and is studying other potential treatments for FOP.
