Applied Therapeutics has announced plans to discuss a potential new drug application (NDA) submission with the US Food and Drug Administration (FDA) after the Phase III trial (NCT05397665) investigating its sorbitol dehydrogenase (SORD) deficiency drug Govorestat (AT-007) had positive interim results.  

Treatment with Govorestat resulted in a statistically significant improvement on the primary endpoint at 12 months – the correlation of sorbitol with the Charcot Marie Tooth functional outcome measure (CMT-FOM), which includes a ten-metre walk-run test, four stair climb, sit-to-stand test, six-minute walk test and dorsiflexion. The Phase III INSPIRE trial enrolled 56 patients aged 16-55 years with SORD deficiency. 

The treatment also demonstrated significant improvements in secondary endpoints, including a statistically significant effect on the CMT health index with various aspects such as lower limb function, mobility, fatigue, pain, sensory function, and upper limb function showing improvement. Applied reported that Govorestat shows a favourable safety profile with no adverse events reported. 

At the 24-month mark, the ongoing INSPIRE trial will reevaluate clinical outcomes, focusing on the ten-metre walk-run test as the primary measure of clinical effectiveness.  

Applied is now requesting a pre-new drug application (NDA) meeting with the FDA’s neurology division for Govorestat, which has both orphan drug and fast track designations. 

SORD deficiency is a genetic condition where the body lacks enough of the enzyme sorbitol dehydrogenase, which is needed to break down sorbitol, a sugar found in many fruits and artificial sweeteners. This deficiency can lead to symptoms such as developmental delay, intellectual disability, and seizures. 

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Govorestat acts as an inhibitor for aldose reductase, which catalyses the reduction of galactose to galactitol. Galactosaemia is characterised by the inability of the body to convert galactose to glucose. The drug candidate acts by inhibiting aldose reductase and alleviates the disease condition. 

According to GlobalData’s Pharma Intelligence Center, Govorestat is forecast to generate $81m in sales in 2029. 

GlobalData is the parent company of Clinical Trials Arena.  

In the announcement accompanying the positive results, Applied chief medical officer Riccardo Perfetti said: “We look forward to meeting with regulatory agencies regarding a path to potential approval based on this data, and endeavour to bring this important treatment to patients as quickly as possible.”