KaloBios receives orphan drug designation for benznidazole in the treatment of chagas disease.
KaloBios Pharmaceuticals, Inc., a biopharmaceutical company focused on advancing medicines for patients with neglected and rare diseases, announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to benznidazole for the treatment of Chagas disease, a neglected tropical disease.
“Chagas disease is a serious, life-threatening and underserved disease in the U.S., and patients suffering from this disease are still without an FDA-approved treatment option,” said Cameron Durrant, MD, chairman and CEO of KaloBios. “This orphan designation highlights another example of our continued execution to swiftly advance the benznidazole program to potentially bring a safe and effective treatment for Chagas disease in the U.S.”
Under the U.S. Orphan Drug Act, FDA’s Office of Orphan Products Development grants orphan drug designation to drugs and biologics defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and provides several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.
KaloBios expects to submit a New Drug Application (NDA) submission for benznidazole in the first quarter of 2018. The company received clearance from the FDA in June for its Investigational New Drug (IND) application. Earlier this year, it also received positive guidance from the FDA supporting the company’s proposed 505(b)(2) regulatory pathway for benznidazole, enabling the company to use data obtained from previously conducted studies to demonstrate safety and efficacy in the treatment of Chagas disease within the U.S.
Benznidazole is an oral anti-parasitic medication used in the treatment of Chagas disease, caused by a protozoan parasite Trypanosoma cruzi carried and transmitted by triatomine insects (often called “kissing bugs”). An estimated 350,000 people in the United States are infected with Chagas disease, which, if left untreated, can lead to serious and potentially life-threatening cardiovascular, gastro-intestinal and neurological complications. According to a May 2017 PLOS Neglected Tropical Diseases paper, those infected have an almost 18 times average higher risk of death from Chagas-related cardiac issues than those not infected. Benznidazole is the current preferred treatment for Chagas disease in other parts of the world but is not currently approved by the FDA in the U.S.
(Source: Globe Newswire)