Posted by Michael Wonder on 07 Aug 2019
ICER issues statement regarding manufacturer's manipulation of data from animal testing of Zolgensma
7 August 2019 - Yesterday afternoon, the FDA announced that Novartis informed the agency in June of a data manipulation issue that affects the accuracy of certain animal testing data related to onasemnogene abeparvovec (Zolgensma, Novartis/AveXis), a gene therapy approved in May to treat certain individuals with spinal muscular atrophy (SMA).
In its announcement, the FDA clarified that "these data do not change the agency's positive assessment of the information from the human clinical trials that were conducted as part of the development program."
The Institute for Clinical and Economic Review (ICER) is deeply concerned about issues related to data integrity, and we are monitoring this situation closely. The animal data identified by the FDA did not factor into ICER's assessment of the comparative clinical effectiveness and economic value of Zolgensma. However, if additional investigations reveal that data from the human trials also were compromised in any way, ICER will issue an update to our earlier assessment.
