Santhera and ReveraGen announce positive results from VISION-DMD study

pharmafile | November 23, 2021 | News story | Manufacturing and Production  

Santhera Pharmaceuticals and ReveraGen BioPharma have announced new topline results after completion of the VISION-DMD study at week 48.

VISION-DMD was a pivotal double-blind Phase IIb study, designed to demonstrate the efficacy and safety of vamorolone compared to placebo and prednisone (active control) in the treatment of DMD. In the first 24 weeks, 121 ambulant boys aged 4 to 7 years were randomised to receive vamolorone, prednisone, or placebo. 114 subjects continued for another 24 weeks, where those on vamorolone continued to the end of the study on these doses. Those on prednisone and placebo had been previously randomised to receive vamorolone after a four week tapering period.

Of the 114 subjects who entered into period two, two subjects discontinued treatment (one adverse event, one withdrawn consent). The safety profile was generally consistent at week 48 compared to the results previously reported at week 24.

Dario Eklund, CEO of Santhera, commented: “Back in June, we announced the pivotal 24-week data from the VISION-DMD study which the FDA recently considered sufficient for our planned NDA filing. Now, we are very pleased to announce the completion of the VISION-DMD study, providing longer term data which both confirm earlier findings but importantly also support the potential benefits of vamorolone in overcoming some of the challenges these young children and families face in tolerating long term use of corticosteroids.

“We look forward to commencing the NDA submission with our partner ReveraGen and working with regulators to making vamorolone available as soon as possible.”

Lina Adams

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