RARE Daily

FDA Places Partial Hold on Applied Therapeutics’ Pediatric Study of Treatment for Rare Metabolic Disorder

August 17, 2020

Rare Daily Staff

The U.S. Food and Drug Administration placed a partial clinical hold on Applied Therapeutics’ ACTION-Kids study of AT-007, the company’s experimental therapy to treat galactosemia, a rare metabolic disorder that prevents people from breaking down a form of sugar in dairy products and other foods.

Galactosemia is a condition caused by a genetic mutation. Depending on the specific mutation, people may have different forms of the disease, which varies in symptoms and severity. The inability to properly breakdown the sugar and convert it to energy leads to cognitive impairment, failure to thrive in infancy, global developmental delay, and liver failure.

AT-007 is a central nervous system penetrant aldose reductase inhibitor (ARI) in clinical development for the treatment of galactosemia. AT-007 has been studied in an animal model of galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications. The FDA previously granted Applied Therapeutics’ AT-007 Orphan Drug and Rare Pediatric Disease designations.

In a filing with the U.S. Securities and Exchange Commission on August 14, the company reported that the FDA placed the partial clinical hold on the ACTION-Kids study and requested it to provide additional technical information to ensure every patient in the study has access to the prospect of direct benefit of the drug.

The ACTION-Kids study is evaluating AT-007 in children age 2 to 17 for safety, pharmacokinetics, and reduction in the toxic biomarker galactitol. The study includes a placebo-controlled dose range finding segment evaluating up to seven days of consecutive dosing to determine the optimal dose in children, followed by a placebo-controlled 90-day study evaluating safety and biomarker efficacy.

The company said the partial clinical hold does not relate to concerns regarding the safety profile of AT-007. The study is currently in the dose range finding segment, and the partial clinical hold does not introduce any disruption to ongoing treatment.

The adult ACTION- Galactosemia study, which is in the long-term extension phase, is not affected by the partial clinical hold.

Applied Therapeutics said it will work closely with FDA to resume the study as soon as possible and plans to submit the supporting technical information requested by the FDA promptly. The FDA has 30 days thereafter to review the information and to notify the company if the ACTION-Kids Study may be resumed.

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