Mixed regulatory progress for rare disease drug leriglitazone

Minoryx Therapeutics, a Spanish biotech specializing in therapies for orphan CNS disorders, will submit for European approval for leriglitazone by the middle of next year.

The move follows positive results from the NEXUS trial, evaluating the drug for cerebral adrenoleukodystrophy children and adults.

The trial showed that 35% of children achieved clinical and radiological disease arrest while on leriglitazone, compared to an expected 10% from natural disease progression.