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FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy

Today, the U.S. Food and Drug Administration (FDA) has granted its approval for Vamorolone, a new treatment for Duchenne Muscular Dystrophy (DMD). Marketed as Agamree, this therapy represents a new milestone in the management of DMD, offering treatment to patients aged 2 years and older.


Read the Announcement


Agamree, previously known as vamorolone, is a dissociative steroid therapy designed to reduce the adverse effects of traditional corticosteroids while retaining their anti-inflammatory benefits. Agamree, through the VISION-DMD study, demonstrated superiority in improving the time to stand test (TTSTAND) velocity compared to a placebo. Earlier this month, the EMA’s CHMP had issued a positive opinion on Vamorolone.


About the Vamorolone study

The VISION-DMD study consisted of a 2-part evaluation of vamorolone, assessing its efficacy, safety, and pharmacokinetics. The study included ambulatory boys aged 4 to 7 years with DMD across 33 trial sites. Results from this study revealed not only the drug’s efficacy in primary and secondary endpoints but also its favorable safety and tolerability profile when compared to prednisone.

In part 2 of VISION-DMD, the open-label portion, the drug demonstrated further improvement on multiple efficacy outcomes. Notably, early-starters on vamorolone 6.0 mg/kg/day showed significant gains in various outcomes compared to delayed-starters, providing promising results.


Vamorolone launch

Catalyst Pharmaceuticals acquired the North American license and commercial rights for vamorolone from Santhera Pharmaceuticals in July 2023. Santhera, the original developer of vamorolone, will transfer the approved new drug application (NDA) to Catalyst. Catalyst Pharmaceuticals is set to launch Agamree in the first quarter of 2024, with a commitment to patient accessibility and affordability. This approval offers renewed hope to DMD patients and potentially opens doors for managing other chronic inflammatory conditions.


Source: https://www.neurologylive.com/view/fda-approves-vamorolone-agamree-duchenne-muscular-dystrophy