Raxone idebenone regulatory update

Santhera said FDA would not support its plan to submit an NDA for Raxone idebenone to treat Duchenne muscular dystrophy (DMD) without data from the double-blind, placebo-controlled, international Phase III SIDEROS trial, which are due in 2H19. Santhera had hoped to seek accelerated approval of Raxone to treat DMD patients who are not using glucocorticoids based on the completed double-blind, placebo-controlled, international Phase III DELOS trial, with SIDEROS serving as a confirmatory trial that could expand Raxone’s label.

In 2014, Santhera said Raxone met DELOS’s primary endpoint. The short-chain benzoquinone led to a 3.1% decline in peak expiratory flow from baseline to week 52 vs. a 9% decline for placebo (p=0.04) in DMD patients not using glucocorticoids (see BioCentury, May 26, 2014). ...