Galactosemia (G) is due to either partial or complete deficiency of the enzyme galactose-1-phosphate uridyl transferase (Ts). In females with G, there is a high incidence of premature ovarian ...
Applied Therapeutics' (APLT) lead drug candidate, govorestat, likely faces "significant" uncertainty in potentially treating galactosemia after the US Food and Drug Administration rejected the ...
The NDA for govorestat for the galactosemia indication was accepted by the FDA in late February under its Priority Review pathway. Year to date, shares of Applied Therapeutics have skyrocketed 155 ...