Why Was It Approved? The FDA approved Itvisma based on results of a study in children and teens with SMA type 2 who could sit but had never walked and had not received any previous treatment.
In the study, those who received Itvisma showed more improvement in muscle control and movement than those who received a sham procedure (a procedure that mimics drug delivery, but without the active ingredient).
Another study looked at how safe and effective Itvisma was in 27 children with SMA in the same age group who had stopped other SMA treatments (nusinersen or risdiplam). After 52 weeks, Itvisma was found to be safe and well tolerated. Motor abilities were maintained, showing that movement skills did not decline as they normally do in SMA. On average, motor function scores showed a slight improvement of 1.05 points, showing that Itvisma can help preserve and even slightly improve movement in children who have previously received other therapies. The most common side effects were fever, stomach symptoms, and upper respiratory tract infections, and these were seen in both the treatment and sham groups. Serious side effects included rare occurrences of increased liver enzymes, disrupted sensation, and vomiting in the Itvisma-treated group.
