Rare Daily Staff

The U.S. Food and Drug Administration granted Applied Therapeutics Fast Track designation to its experimental therapy AT-007 for the treatment of galactosemia, a rare metabolic disease.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for accelerated approval and priority review if relevant criteria are met.

People with Galactosemia have mutations or deletions in their DNA that are passed down from family members. The DNA alterations can lead to different subtypes of galactosemia, such as GALT Deficiency (known as classic galactosemia) or GALK Deficiency. Galactose is a sugar found in foods, but the human body also naturally produces galactose on its own. Because of that, diet alone cannot prevent the long-term consequences of galactosemia. A treatment for those living with galactosemia is needed. Aldose reductase (AR), an enzyme known to play a role in many diseases including galactosemia, converts galactose into galactitol, a toxic metabolite that builds up in tissues and organs and can cause long-term disease complications.

AT-007 is a central nervous system penetrant aldose reductase inhibitor in development for the treatment of several rare diseases. In an animal model of galactosemia, AT-007 reduced toxic galactitol levels and prevented disease complications. AT-007 significantly reduced plasma galactitol levels vs. placebo in adults with galactosemia in a phase 2 registrational trial; the long-term extension in adults remains ongoing.

AT-007 is currently being studied in a registrational trial in children ages 2-17 with galactosemia. AT-007 is also in clinical development for the treatment of SORD Deficiency, a rare progressive hereditary neuropathy, and in development for the treatment of phosphomannomutase 2 deficiency (PMM2-CDG), a congenital disorder of glycosylation.

AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the FDA for the treatment of galactosemia and PMM2-CDG.

“Galactosemia is a devastating rare metabolic disease that progressively worsens over time and greatly impacts patient quality of life,” said Shoshana Shendelman, founder and CEO of Applied Therapeutics. “Fast Track designation offers several important opportunities to work closely with the FDA through the review and approval process to ensure that AT-007 is available to patients as quickly as possible.”

Photo: Shoshana Shendelman, founder and CEO of Applied Therapeutics

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