Risdiplam 

Risdiplam has received FDA Priority Review and they will make a decision by May 24, 2020. Specifically, this approval will be for a broad label of the drug which will include SMA patients with the most severe form of the disease. Currently, the drug is only approved for older patients who do not have a severe form of disease.

Last year Roche submitted to the FDA results from both the SUNFISH trial and the 1st part of the FIREFISH Trial. In the SUNFISH trial participants were between the ages of 2 and 25.

Roche is also investigating the effects of this therapy in SMA patients who had previously received a different treatment and in young babies who have received the SMA diagnosis but haven’t yet begun showing symptoms.

Full results from the SUNFISH and FIREFISH trial will be announced soon. Although Roche admits that they expect the most dramatic benefits to come for the less severe patients, preliminary results released from the FIREFISH trial document the impact this drug could have on children with the most severe form of the disease. 41% of patients were able to sit unassisted just one year after starting the treatment.

As the final results from these trials are released, we will get a better idea of how risdiplam compares to spinraza and zolgensma. But hopefully, SMA patients with all forms of the disease will have a new therapeutic option soon.