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Applied Therapeutics' metabolic disorder drug gets US FDA orphan designation

Applied Therapeutics Inc. said the U.S. Food and Drug Administration has granted orphan-drug designation to its treatment for a rare pediatric metabolic disease that has no known cure or approved therapy.

The New York-based biotechnology company is developing AT-007 as a potential treatment for galactosemia, a metabolic disorder that affects the body's ability to process a sugar called galactose. High levels of galactose are converted by the body into a toxic sugar alcohol called galactitol. Galactose is produced naturally by the human body, so dietary restrictions cannot prevent complications of the disease. Long term effects of the disease include cataracts in the eye, delayed development and growth, intellectual disability, liver disease and kidney problems.

About 2,800 patients in the U.S. have galactosemia, the company said in a May 28 press release.

The FDA grants orphan-drug designation to therapies for rare disorders that affect fewer than 200,000 people in the U.S. Incentives include seven years of market exclusivity after approval, the exception of fees for certain drug indications and tax credits for qualified clinical trials.

Applied Therapeutics plans to start a phase 1/2 clinical study of AT-007 later in the year. The drug reduced galactitol levels and prevented disease complications during pre-clinical studies in animal models.