The P-III (INSPIRE) trial evaluates Govorestat (QD) vs PBO in patients (n=56) with SORD deficiency aged 16-55yrs. across the US & EU. The 1EPs was correlation of sorbitol with composite clinical outcomes & 2EP was effects on CMT-HI
The 12mos. interim analysis depicted a statistically significant correlation between sorbitol level & prespecified CMT-FOM, sustained reduction in sorbitol level & highly significant effects on the CMT-HI. Based on these results, the company plans to submit an NDA to the US FDA
Govorestat is a CNS penetrant ARI being developed for the treatment of various neurological diseases incl. Galactosemia, SORD Deficiency & PMM2-CDG. Govorestat received the OMPD (EMA) & ODD, PRDD & FTD (US FDA)
Ref: Applied Therapeutics | Image: Applied Therapeutics
Related News:- Applied Therapeutics Reports P-III trial (INSPIRE) Results of AT-007 for Sorbitol Dehydrogenase Deficiency
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