The FDA has approved a new medicine called Sohonos (palovarotene). It is meant to help people with a rare genetic condition called fibrodysplasia ossificans progressiva, or FOP. In FOP, the body slowly forms extra bone in places where bone should not be, such as muscles and soft tissues. This can lead to lasting disability over time.
Sohonos is approved to reduce the amount of new extra bone that forms. It is for females aged 8 years and older and males aged 10 years and older. The medicine is taken daily, and the dose can be raised for a short time during flare-ups, which are periods when the condition worsens. Younger children receive doses based on their weight. Females who could become pregnant must have a negative pregnancy test before starting.
This approval gives patients and families a new treatment option for a condition that has had very few. However, the label does not include data from clinical trials, so the full picture of benefits and risks is not yet clear from the label alone. Patients should talk with their doctor to understand if Sohonos is right for them and to discuss possible side effects.
