According to a story from prnewswire.com, the biopharmaceutical company Aridis Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to the company’s investigational product AR-501, which is being developed to treat lung infections in patients with cystic fibrosis. Aridis is focused on the development of targeted immunotherapies to treat potentially life threatening bacterial infections.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
About Orphan Drug Designation
Orphan Drug designation is usually reserved for treatments that are intended to treat a disease that is considered rare, which is defined as any illness or condition that affects less than 200,000 people in the US annually. To qualify, an experimental drug must display advantages in safety or efficacy over current therapies or must fulfill a medical need that no current therapy meets. This designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity of the drug is approved.
About AR-501
AR-501 is not an antibiotic but is instead an inhaled formulation of gallium citrate. The drug antagonizes several pathways that are dependent on iron, of which gallium serves as an analog. These pathways are critical for the survival of bacteria. Prior studies have shown that AR-501 can work in combination with many antibiotics, but since it is not an antibiotic, it is also effective against strains of bacteria that have developed antibiotic resistance.
