Appili Therapeutics Inc., an anti-infective pharmaceutical development company, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for ATI-1501, a taste-masked reformulation of metronidazole with the potential to effectively treat Clostridium difficile infection (CDI) in children.
CDI is one of the U.S. Centers for Disease Control’s most urgent antibiotic-resistant bacterial threats. CDI affects over half a million Canadians and Americans each year. It reoccurs in 83,000 patients and is responsible for 29,000 deaths annually. The incidence of CDI in children has been steadily increasing over recent years, and thus a taste-masked metronidazole formulation is urgently needed for this population.
“The bitter metronidazole tablet is highly unpalatable for most children, so ATI-1501 has the potential to fundamentally improve compliance rates for these patients who are in need of a safe and effective treatment for CDI,” said Kevin Sullivan, CEO of Appili Therapeutics.
The FDA Orphan Drug Act provides orphan status to new drugs that are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect less than 200,000 people in the United States. This orphan drug designation provides Appili with several benefits, including seven years of marketing exclusivity upon approval of ATI-1501, reduced FDA application and administration fees and tax credits for clinical research in the U.S.
“In just three months, we received orphan drug designation from the FDA,” continued Mr. Sullivan. “Having this quick turnaround is an indication of the high quality of the regulatory team we have built, and an important step on our path to becoming the first company to offer an approved treatment for C. difficile designed specifically for children affected by this serious infection.”
About Appili Therapeutics
Appili is dedicated to identifying, acquiring and advancing novel therapeutics in the area of infectious disease and has commenced development on two anti-infective programs, while continuing its search to acquire additional high quality anti-infective programs at all stages of development. The company plans to take its lead drug candidate, ATI-1501, a potential treatment for C. difficile infection, into clinical trials by 2017. Appili’s second product, ATI-1503, is a novel antibiotic with the potential to treat deadly infections such as Klebsiella pneumoniae. These drug-resistant Gram-negative bacterial infections have been identified by the U.S. Center for Disease Control (CDC) as posing the highest threat to human health. Appili is co-founded and backed by the life-science/healthcare investment bank Bloom Burton & Co. For more information visit www.appilitherapeutics.com.