Applied Therapeutics Announces FDA Orphan Drug Designation for AT-007

Applied Therapeutics (NASDAQ:APLT) has announced that the US Food and Drug Administration (FDA) has granted the company orphan drug designation for AT-007 for late stages of preclinical development to treat Galactosemia.

As quoted in the press release:

“We are pleased to obtain Orphan Drug Designation for AT-007 in Galactosemia, a devastating rare metabolic disease for which there are no approved treatments,” said Shoshana Shendelman, PhD, Founder, Chief Executive Officer and Chair of the Board of Applied Therapeutics. “Galactosemia patients are in desperate need of treatment options, and we are committed to advancing AT-007 into clinical development as quickly as possible due to the urgent unmet medical need.  We look forward to taking advantage of the opportunities that Orphan Designation provides in order to bring this potential new treatment option to patients and families as rapidly as possible.”  Applied Therapeutics plans to initiate a Phase 1/2 biomarker based clinical study with AT-007 in Galactosemia patients later this year.

The FDA Office of Orphan Products Development grants Orphan Drug Designation to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States. The designation allows Applied Therapeutics to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; exemption from FDA application fees for Galactosemia; and tax credits for qualified clinical trials.

Click here to read the full press release.