New Oral Drug for SMA may Help Young Children with the Most Severe Form of the Disease

Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to three for all forms of disease.

Current Options

Spinraza was first approved in 2016 as a therapeutic option specifically for SMA. Then in 2019 Zolgensma was approved. This is a gene therapy which is meant to treat young children.

Roche has been working on a new therapeutic option called risdiplam. It’s unique in the fact that its an oral therapy, meaning patients don’t have to deal with injections. Spinraza can only be administered through the spinal cord, directly into the cerebrospinal fluid. Zolgensma has also been administered this way in some trials. An oral therapy means patients don’t have to face this invasive procedure.


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.

You can read more about this therapy here.

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