Like prednisone and deflazacort, vamorolone will lead to adrenal insufficiency, i.e. because an external steroid is provided, the adrenal glands stop producing cortisol. This means that just like with other steroids, you need to be weaned off vamorolone and in case of an emergency (fracture, sickness) you need stress dosing to allow the body to cope (stress dosing can be with prednisone or deflazacort). So the @Bpn9211Brian PJ protocol for stress dosing applies to vamorolone just as much as to 'regular' steroids!
Vamorolone was developed by @ReveraGen but is currently licensed to @Santhera In North America, Catalyst Pharmaceuticals has a license, in Greater China Sperogenix Therapeutics has a license.
The author describers preclinical and clinical tests with vamorolone: In Duchenne and Becker mouse models vamorolone reduced pathology and was bone sparing. In all models and Duchenne patients vamorolone reduced inflammatory markers. In Duchenne patients vamorolone also has a positive effect on muscle function (time to stand).
This effect was significant compared to placebo and similar to prednisone. Vamorolone is metabolized by the liver and excreted via urine and feces. Vamorolone also leads to steroid like side effects such as weight gain and behavioral issues. No growth stunting has been observed also so far, bone mineral density seems to be better than with steroids. Additional trials with vamorolone are ongoing in patients with Becker muscular dystrophy. Vamorolone is approved by @US_FDA for Duchenne patients >2 years and by @EMA_News for Duchenne patients >4 years old.
Of course we do not know yet what will happen after long term use of vamorolone: will it be as effective in slowing down progression, loss of ambulation delay and reducing respiratory function loss? And will the reduced side effects persist after long term use?
These are data that have to be collected now that the drug is approved.
Vamorolone development was sponsored in large parts by the Duchenne patient community and also in collaboration with the patient community (e.g. they were part in the EU funded VISION-DMD project @Vision_DMD)
