This week, Vertex Pharmaceuticals Incorporated, a global biotechnology company that aims to discover, develop and commercialize innovative new medicines for diseases such as cystic fibrosis, announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the use of KALYDECO (ivacaftor) in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
CF is a chronically progressive life-threatening disease caused by a genetic mutation that disrupts the body’s ability to hydrate and effectively clear mucus in the lungs and/or digestive tract. According to the CDC, in the US, approximately 1,000 new cases are diagnosed each year with more than 75% of those patients under the age of two. An estimated 30,000 children and adults in the US and 70,000 worldwide have the disease. Patients diagnosed with CF have a lifespan of approximately 30 years, with many patients living into their forties.
The genetic mutation that causes CF affects the normal functioning of the lungs and digestive system by creating abnormal amounts of very sticky mucus that covers the lungs, pancreas, and other important organs of the respiratory and digestive systems. This sticky mucus attracts foreign pathogens, such as viruses and bacteria, making patients more susceptible to infectious diseases (i.e. pneumonia). The high microbial burden causes inflammation of the lung tissue and a high likelihood of tissue destruction due to the frequency of infections.
The FDA granted the sNDA based on preclinical data provided by Vertex from a Phase 2a clinical trial showing that in 19 of the 24 patients with 23 residual function mutations, 8 of the 23 mutations proposed in the sNDA were represented.
In a company press release announcing the FDA’s decision, Dr. Jeffrey Chodakewitz, MD, Executive Vice President and Chief Medical Officer at Vertex, stated,”Given the severity of cystic fibrosis, we are committed to getting KALYDECO to more people as quickly as possible. Based on the established safety profile of KALYDECO and our increasing understanding of the biology of these specific residual function mutations and their response to ivacaftor, we believe that people with these mutations would benefit from treatment with this medicine.”
The FDA has set a target review date of Vertex’s KALYDECO data for February 6, 2016.