FDA approves Santhera's DMD drug Agamree

The US Food and Drug Administration (FDA) yesterday approved the new drug application (NDA) for Agamree (vamorolone) as a treatment of patients aged two years or more with Duchenne muscular dystrophy (DMD), that was submitted by Swiss drugmaker Santhera Pharmaceuticals (SIX: SANN), whose shares were down 4.8% at 12.66 francs in mid-morning trading.

The FDA approved Agamree on the basis of the pivotal Phase IIb VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, Agamree was administered at doses ranging from 2 to 6mg/kg/day, extending for a period of up to 48 months.

Santhera noted that, compared with current standard of care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory and behavior. The studies in the development program were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries.