At the time of designation, one medicine, Spinraza, was authorised for the treatment of spinal muscular atrophy. Patients also received supportive treatment to help them and their families cope with the symptoms of the disease. This included chest physiotherapy and physical aids to support muscle function, and ventilators to help with breathing.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with spinal muscular atrophy. Early data showed that branaplam improved muscle function and reduced the need for assisted feeding and ventilation. Also, branaplam is given by mouth while Spinraza is given by intrathecal injection (into the lower back, directly into the spine).

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.