FDA’s oncology advisory committee backs potential high-risk neuroblastoma treatment

The FDA’s On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee on Wednes­day vot­ed 14 to 6 in fa­vor of the ef­fi­ca­cy of US WorldMeds’ po­ten­tial ther­a­py to re­duce the risk of re­lapse in pe­di­atric pa­tients with high-risk neu­rob­las­toma.

The vote sets up a tricky and po­ten­tial­ly prece­dent-set­ting de­ci­sion for FDA, as ad­comm mem­bers ques­tioned both FDA and the spon­sor over why a ran­dom­ized clin­i­cal tri­al (RCT) for the ther­a­py, known as eflor­nithine or DF­MO, was not run. FDA rec­om­mend­ed an RCT back in 2015, but US WorldMed in­sist­ed that its ex­ter­nal­ly con­trolled tri­al re­sults are enough to sup­port the use of eflor­nithine in this hard-to-treat pop­u­la­tion of those who have al­ready re­ceived oth­er ther­a­pies.

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