AAN 2026 | Unmet needs and emerging therapies in pediatric NMOSD
Kelsey Poisson, MD, Nationwide Children’s Hospital, Columbus, OH, discusses the key unmet needs in the management of pediatric neuromyelitis optica spectrum disorder (NMOSD), highlighting the lack of FDA-approved therapies for children. Dr Poisson notes that there are ongoing clinical trials for pediatric NMOSD, including those for ravulizumab, inebilizumab, and satralizumab, which are open-label and aim to provide access to these medications for children. This interview took place at the 78th American Academy of Neurology (AAN) Annual Meeting in Chicago, IL.
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Transcript
There are so many and I think that really is due to a lot of reasons but mostly because of how rare this disorder is in children and so only three to five percent of cases of NMOSD are seen in children. And so the research is really lacking in terms of having robust prospective studies that tell us where we should go with treatment or how kids will do. And so I think that really the biggest unmet need, though, overall, is the fact that we have no FDA-approved therapies in the US for pediatric NMOSD, whereas on the adult side, we now have four, which is wonderful...
There are so many and I think that really is due to a lot of reasons but mostly because of how rare this disorder is in children and so only three to five percent of cases of NMOSD are seen in children. And so the research is really lacking in terms of having robust prospective studies that tell us where we should go with treatment or how kids will do. And so I think that really the biggest unmet need, though, overall, is the fact that we have no FDA-approved therapies in the US for pediatric NMOSD, whereas on the adult side, we now have four, which is wonderful. And so really the main hurdle is getting these clinical trials to get enough recruitment of enough children to be able to complete these studies so we can get FDA-approved treatments. And so there are currently three clinical trials ongoing for children with aquaporin-4 positive NMOSD, and really they’re targeting only about 15 children to be enrolled, but just given how rare this disorder is and that we have decent off-label medications we can use that a lot of centers are very familiar with and comfortable with, you know, those trials have not reached enrollment yet but are getting closer. The clinical trials that are ongoing for children are for ravulizumab or Ultomiris. Children were actually previously enrolled in the eculizumab trial but because that medication is dosed every two weeks, and most children are not a huge fan of getting IVs or missing school every two weeks, those patients were transitioned to the ravulizumab trial because, again, we’re trying to get enough children to get to FDA approval and access for these medications. So there’s that trial, there’s the inebilizumab or Uplizna trial, and then there’s also the satralizumab trial. And so each of these trials are, again, only going to get to a small number of patients, but they’re open label. And so all children would have access to the drug in these trials because it’s no longer ethical to use placebo for NMO just because of how disabling relapses can be. And children were actually enrolled in the original satralizumab trial. And so there were a few children in that trial down to age 12. And so that’s why satralizumab is actually approved in certain countries, including Canada. But the US, because of the low enrollment, did not give FDA approval for that medication.
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