History lesson

CHMP's recommendation for conditional approval of Translarna ataluren to treat Duchenne muscular dystrophy just five months after rebuffing the compound suggests the agency is getting a clearer picture about how to interpret efficacy measures across the course of the disease.

In January's negative opinion, EMA's CHMP noted PTC Therapeutics Inc.'s main study of Translarna - a 174-patient Phase IIb trial (Study 007) - did not show a benefit on the primary endpoint of six-minute walk distance (6MWD). The committee also said other measures of effectiveness provided "only limited supportive evidence of the beneficial effects of Translarna."...