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Dr Migvis Monduy Explains What Sets Agamree Apart From Other Corticosteroids for DMD

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Vamorolone (Agamree) is a first-in-class corticosteroid to treat Duchenne muscular dystrophy (DMD) in patients 2 years and older.

Migvis Monduy, MD, pediatric neurologist and neuromuscular specialist at Nicklaus Children's Hospital, discusses the unique mechanism of action and reduced adverse effects of vamorolone (Agamree) compared with traditional corticosteroids, and how it offers promising benefits for prolonging ambulation and mobility in patients with Duchenne muscular dystrophy (DMD).

Transcript

How does vamorolone’s mechanism of action differ from traditional corticosteroids in treating DMD?

Vamorolone is still an anti-inflammatory, like the other corticosteroids. It inhibits NF-κB receptors in the same way but with less transactivation, which leads to a better side effect profile. It's been known as a dissociative steroid, meaning that it retains the efficacy without the side effects. One other difference is the molecular structure—it lacks 11β-hydroxy carbonyl moiety on the steroidal C ring, thus it loses a site of action for an 11β-hydroxysteroid dehydrogenase enzyme, which is the one that mediates the bone side effects. It is also a potent mineralocorticoid receptor antagonist as opposed to an agonist like the other corticosteroids.

How does vamorolone prolong ambulation and mobility for patients with DMD?

We know that long-term data for corticosteroids [show], in general, they do prolong ambulation. And it's a reason why we prescribe these medications to boys with DMD—we want them to retain their mobility as long as possible and also delay the onset of scoliosis or cardiomyopathy down the line.

We don't have long-term data on vamorolone yet, but in the short-term data from the VISION-DMD study, it does show that over the short term, it is just as effective as prednisone, so we hope that in the long term, that will translate to the same sort of long-term outcomes. Hopefully even better, because a lot of times, because of side effects, patients are not able to tolerate full doses of prednisone, and then you have to adjust the dose or decrease it, so they're not on the most effective dose.

Hopefully, having a steroid that has less side effects can lead to the patient staying at the effective dose for longer periods of time, and then that would translate into those long-term benefits of prolonging mobility and maintaining that mobility over the long term.

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