FDA Okays Drug for Duchenne Muscular Dystrophy

The US Food and Drug Administration (FDA) has approved vamorolone oral suspension (Agamree, Santhera) for the treatment of Duchenne muscular dystrophy (DMD) in patients as young as age 2 years, the company has announced.

Vamorolone is a structurally unique steroidal anti-inflammatory drug that potently inhibits proinflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor.

“Corticosteroids have been a first line treatment for DMD for many years but their utility has always been limited by the side effect profile, which includes weight gain, short stature, and decreased bone density among others,” Sharon Hesterlee, PhD, chief research officer for the Muscular Dystrophy Association (MDA), said in a statement.

The approval of vamorolone “provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some negative side effects associated with corticosteroids,” Hesterlee added.

The approval was based on data from the phase 2b VISION-DMD study, supplemented with safety information collected from three open-label studies.

Vamorolone was administered at doses ranging from 2 to 6 mg/kg/d for a period of up to 48 months.

Vamorolone demonstrated efficacy similar that of traditional corticosteroids, with data suggesting a reduction in adverse events ― notably related to bone health, growth trajectory, and behavior.

Vamorolone had received orphan drug status for DMD, as well as fast track and rare pediatric disease designations. It will be made available in the US by Catalyst Pharmaceuticals.

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