Important safety update on SMN2 splice-modifying drug, RG7800

15 May 2015

Roche, PTC Therapeutics, and the SMA Foundation have provided a critical update on the safety of their SMN2 splice-modifying compound, RG7800, which recently entered a Phase Ib/IIa clinical trial in adults and children with SMA (click here for more information).

Due to safety concerns, administration of RG7800 in the trial named Moonfish has been immediately suspended as a precautionary measure. In long-term experiments in mice, unexpected side-effects have been reported in the eyes of treated animals.

As standard in the development of new drugs, RG7800 was being tested over an extended period of time in order to highlight any potential safety issues due to prolonged exposure to the drug.

The companies involved in the development of RG7800 immediately conducted an internal review of safety data from all patients involved in the trial, and no problems were identified. It should also be noted that the concentration of drug being tested in the mice was higher than that being used to treat patients in the trial.

The findings of the animal study are being thoroughly examined, and further information is to be provided by the involved parties in the near future.

Further Information

Update on Clinical Development of RG7800