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BioWorld - Saturday, May 16, 2026
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Home » Live by the SORD: Applied Therapeutics looks to rare disease NDA
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Live by the SORD: Applied Therapeutics looks to rare disease NDA

Feb. 15, 2024
By Lee Landenberger
Based on positive phase III study data, Applied Therapeutics Inc. plans to take its CNS-penetrant aldose reductase inhibitor to the U.S. FDA to talk about an NDA for treating the rare disease sorbitol dehydrogenase (SORD) deficiency. Interim data from 12 months of treatment showed govorestat (AT-007) hit the study’s primary endpoints along with several key secondary endpoints. The double-blind, placebo-controlled registrational study of patients ages 16 to 55 is ongoing, with another 12 months of data yet to come. SORD, a hereditary axonal neuropathy created by sorbitol dehydrogenase gene mutations, affects about 3,300 people in the U.S. and about 4,000 in Europe, according to Applied Therapeutics.
BioWorld Clinical Neurology/psychiatric Rare disease

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