FDA challenges safety, efficacy of Geron’s imetelstat ahead of advisory committee meeting

The FDA said Tues­day that it is un­con­vinced the ben­e­fits of Geron’s ime­tel­stat out­weigh the risks in cer­tain ane­mic myelodys­plas­tic syn­drome (MDS) pa­tients who are de­pen­dent on blood trans­fu­sions.

Reg­u­la­tors raised a list of safe­ty and ef­fi­ca­cy con­cerns in brief­ing doc­u­ments pub­lished ahead of Thurs­day’s On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee meet­ing, in­clud­ing that the on­ly ran­dom­ized ef­fi­ca­cy tri­al for ime­tel­stat en­rolled just 13 pa­tients in the US.

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