Setback for galactosemia treatment hopes after US FDA meeting

4 January 2022

Shares in New York, USA-based rare disease firm Applied Therapeutics (Nasdaq: APLT) have fallen by a third, after the company said it would put a US regulatory bid on hold.

The company has been developing AT-007, an aldose reductase inhibitor (ARI), for the treatment of the rare genetic metabolic disease galactosemia.

The condition, for which there is no approved therapy, causes neurological complications including deficiencies in speech, cognition, behavior and motor skills.

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