NEW YORK - The U.S. Food and Drug Administration (FDA) has extended its review of the New Drug Application (NDA) for Applied Therapeutics Inc .'s (NASDAQ:APLT) govorestat, a treatment for Classic Galactosemia, by three months. The new Prescription Drug User Fee Act (PDUFA) target action date is now set for November 28, 2024.
Applied Therapeutics, a clinical-stage biopharmaceutical company, announced that the extension is due to the FDA requiring additional time to review supplemental analyses of data submitted by the company. This additional information has been classified as a Major Amendment to the NDA.
Govorestat (AT-007), an investigational Aldose Reductase Inhibitor (ARI), has been studied in children and adults with Galactosemia and has shown a rapid and sustained reduction in galactitol, a toxic metabolite. The drug's development is significant as it could become the first approved medication for Galactosemia, a rare genetic metabolic disease that leads to neurological complications, juvenile cataracts, and ovarian insufficiency in women.
Despite the delay, Applied Therapeutics remains confident in the potential approval of govorestat and continues to provide the drug to patients with Galactosemia through an expanded access program.
The company's CEO, Shoshana Shendelman, PhD, expressed continued commitment to working closely with the FDA throughout the review process. Govorestat has also been submitted for review to the European Medicines Agency (EMA), with a decision expected in the fourth quarter of 2024.
InvestingPro Insights
As Applied Therapeutics Inc. (NASDAQ:APLT) navigates the FDA's extended review period for its New Drug Application for govorestat, investors and industry observers are closely monitoring the company's financial health and market performance. According to real-time data from InvestingPro, Applied Therapeutics has a market capitalization of $720.03 million, signaling a moderate size within the biopharmaceutical sector. Despite the company's innovative efforts in drug development, it currently operates with negative gross profit margins, as indicated by a gross profit margin of -439.43% for the last twelve months as of Q4 2023.
Investors should note that the stock has demonstrated high price volatility, which could be a factor to consider when evaluating the risk associated with the company's shares. This characteristic is further underscored by a significant 735.37% return over the last year, reflecting both the potential rewards and risks in the biotech industry. Additionally, the InvestingPro Tips highlight that Applied Therapeutics is not expected to be profitable this year, and its short-term obligations exceed its liquid assets, which may influence the company's financial flexibility in the near term.
For those considering an investment in Applied Therapeutics, there are more InvestingPro Tips available that could provide deeper insights into the company's financial position and market potential. Currently, there are 11 additional tips listed on InvestingPro, which can be accessed through the dedicated company page at https://www.investing.com/pro/APLT. To further assist investors in their research, InvestingPro is offering an exclusive 10% discount on a yearly or biyearly Pro and Pro+ subscription with the coupon code PRONEWS24.
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