Santhera Pharmaceuticals Announces AGAMREE® Approval for Duchenne Muscular Dystrophy Treatment
Wednesday, December 11, 2024
Santhera Pharmaceuticals has announced that the Chinese National Medical Products Administration (NMPA) has approved AGAMREE® (vamorolone) for patients aged 4 years and older in China.
AGAMREE is a groundbreaking therapy with a unique mechanism of action. It binds to the same receptor as glucocorticoids but modifies the receptor's downstream activity, distinguishing itself from traditional corticosteroids.
This distinctive mechanism enables AGAMREE to deliver therapeutic benefits while reducing safety concerns typically associated with steroids, positioning it as a dissociative anti-inflammatory and a novel alternative to standard corticosteroids for children and adolescents with Duchenne muscular dystrophy (DMD).
In the pivotal VISION-DMD study, AGAMREE achieved its primary endpoint by significantly improving the standing-to-stand speed (TTSTAND) compared to placebo (p=0.002) after 24 weeks of treatment. It also demonstrated a favourable safety and tolerability profile, with the most common adverse events being Cushingoid appearance, vomiting, weight gain, and irritability—generally mild to moderate in severity.
AGAMREE marks a milestone as the first and only approved treatment for DMD in China. This approval follows the acceptance of the New Drug Application (NDA) by the NMPA in March 2024. The NDA highlighted AGAMREE’s clinically significant safety benefits, including the maintenance of normal bone metabolism, density, and growth, compared to traditional corticosteroids.
DMD is a debilitating condition that affects over 70,000 individuals in China. Until now, there were no approved treatment options for this patient population, making AGAMREE's approval a significant advancement in addressing this critical unmet need.
Source: globenewswire.com
