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Investigational nipocalimab reduced serum immunoglobulin G (IgG), improved functional scores, and was well tolerated in adolescents with generalized myasthenia gravis, according to data from an open-label study presented at the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting.

In this exclusive 妖姬直播 video, Jonathan Strober, MD, of the UCSF Benioff Children's Hospital in San Francisco, discusses the significance of the findings for pediatric patients.

Following is a transcript of his remarks:

The study looked at patients from 12 to 18 years of age, in an open-label study, looking at the safety and efficacy of nipocalimab in this age group. We were able to show that nipocalimab was well tolerated with really very little side effects. In fact, nobody dropped out because of side effects and patients did not have any serious adverse events.

Nipocalimab did what it was shown to do in adult populations, which was lower the IgG levels. And then we were also able to look at functional data looking at QMG [Quantitative Myasthenia Gravis] and MG-ADL [Myasthenia Gravis Activities of Daily Living] scores to see how it would work for these pediatric patients. And similar to what we saw in adult patients, we did see a lowering of scores, both of the scores, with very limited disability at the end of 24 weeks in our patients.

At the time of the study, there were seven patients that had begun to be enrolled. Five of them were included in the analysis since they had received at least one dose of nipocalimab by the time the data was locked. And so it was based on those five patients. And since then, four of those five patients have gone on to continue to get nipocalimab in the open-label extension program.

And we have started the program for younger patients all the way down to 6, and even as low as 2 in some countries, to see if the dosing that we assume is the correct dose based on what we're seeing in adolescents and adults would be the appropriate dosing. And also to make sure that it does the same thing and is safe for these patients.

In pediatrics, we've always been used to using medications that were approved for adults and based on adult dosing, so we never really had studies that showed that. So this is really a game changer for us in pediatric population.

The FDA and the EU have told these companies that they need to do a pediatric arm to get approval, and not just for this drug but for a lot of the drugs that are out there. So it's been really helpful, and I think we know in pediatrics that kids are not just small adults. There are definitely differences and there's different struggles in doing research in some of these patients. It's a much rarer disease in pediatrics, so it's always hard to do, as well. And of course, some of the testing that we do, some of the younger kids have a hard time participating in.

So it's really been great to get these trials done to show that we can do trials in this patient population and that we're confirming dosing in this way. Also, insurance companies who often give us a hard time when drugs are only approved for certain age groups, in using this for younger patients, we will have the data to show the insurance companies that it should be approved for younger patients as well.

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