Experience with Risdiplam and Unmet Needs in SMA


James Wymer, MD discusses his firsthand experience with risdiplam and identifies a key unmet need in the treatment of spinal muscular atrophy.

James Wymer, MD: When risdiplam came out, it gave us a wonderful option. To date, we had had 1 medication that was FDA approved that required the intrathecal therapy and was clearly showing significant benefit. The introduction of an oral medication for patients who have complex spinal disease and were unable to get the intrathecal infusions, this gave them an option. Now we have a way of treating the disease. For other patients who had difficulties with the intrathecal injections for various reasons, this gave them a way, because the most important thing I always feel is that a patient with SMA [spinal muscular atrophy] needs to be treated with these medications to improve. These medications have an impact on the disease, and whether you go with the intrathecal or the oral, the competition is the patients who are not taking medications. We need to get people on these medications. By having an oral option for those who can’t do the intrathecal or for those who choose not to, this gives them another option. It is very well tolerated. Most patients I’ve given it to have done very well. I’ve had some infections, some GI [gastrointestinal] [adverse] effects, but we’ve been able to work with patients to try to get those addressed. There have been one or two with whom we’ve had to have the discussion about switching, but again, this is a conversation. Trying these different options, choosing which one’s going to be the best is something that as a neurologist, you develop a relationship with the patient, you know what’s going to work. And in those patients who may be particular for the oral, this does bring up a perfect option that can allow them to keep an active life, a level of activity, because this is just an oral medication you have to do. You don’t have to worry about the hospital presentation, the intrathecal injections.

The biggest unmet need in treating adult SMA has to do with disease education. We’ve got wonderful medications that can impact the disease, that can slow the decline. I’ll describe a different patient from the case, but somebody who basically as an adult developed a way to compensate for the SMA, so they weren’t interested in treatment because they had their power chair, their apartment, their job, but they came to me because after a while they started declining. Our biggest unmet need is educating these patients about the treatment before that decline occurs because if we can impact and slow that decline and then bring all the other multidisciplinary approaches to it, that person who is comfortable with where they are, we could improve their function and keep them at that functional level for a lot longer with the introduction of these medications.

Transcript Edited for Clarity

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