Although a number of advancements have been made for cystic fibrosis (CF) therapies, there are still many unmet needs for this community. CF is defined as an orphan disease, and as such it provides little financial incentive for pharmaceutical companies to treat or prevent it. Orphan diseases are considered rare in the population and affect fewer than 200,000 individuals.
Vertex’s Kalydeco is the first drug available that is able to treat the underlying cause of cystic fibrosis (CF). It targets a faulty gene and its protein known as the cystic fibrosis transmembrane conductance regulator (CFTR). The FDA has approved Kalydeco for individuals six and older with the G551D mutation of CF. The drug is formerly known as VX-770 and goes by the generic name ivacaftor. Kalydeco (ivacaftor) works by increasing the function of the CF protein, thereby preventing some of the build-up of thick mucus in the lungs. However, Kalydeco doesn’t treat the most common form of CF.
In an effort to continue to develop viable therapies for the disease, Vertex Pharmaceuticals has announced that their investigational drug VX-661 has received FDA Orphan Drug Designation (OOD) for the treatment of CF. VX-661 is a CFTR corrector and is being studied in combination with Kalydeco for patients who have the F508del mutation. VX-661 is currently recruiting participants for a Phase II clinical trial to evaluate the safety and efficacy of VX-661 in combination with Kalydeco in patients with CF who are homozygous (have two copies) for the F508del CFTR mutation. At this time, Vertex Pharmaceuticals has global rights to develop and commercialize VX-661. This treats the most common form of CF (two copies of the F508del mutation), which accounts for approximately 40 percent of the total CF population. Currently, Vertex is studying CFTR potentiator Kalydeco in combination with the CFTR corrector VX-809 (Lumacaftor) and CFTR potentiator Kalydeco in combination with the CFTR corrector VX-661. Vertex received two breakthrough therapy designations (BTDs) for Kalydeco in early 2013, as a monotherapy and in combination with Lumacaftor (VX-809).
In early 2014, Vertex was granted a third BTD for Kalydeco in combination with VX-661 for CF patients who have two copies of the F508del mutation.
Vertex is in the process of identifying additional CF treatment regimens. They have already identified next-generation correctors and are focused on the accelerated discovery and development of correctors that could play a role in a variety of future combination therapies, including a dual corrector approach.
Importance of Clinical Trial Participation
Patients should be aware that the Clinicaltrials.gov website was launched in 2000. It is a free online site to help the public learn more about clinical trials by providing descriptions, locations and other important information about more than 100,000 clinical trials. Although this site has been somewhat successful in engaging more patients into clinical trials, there are still difficulties that can delay or even thwart efforts to move scientific discoveries from lab to the clinical scene. If clinical trials are to be successful, more people need to get involved. Signing up for a clinical trial will benefit medical research and help future generations. In many cases, participants gain personal advantages, such as improved disease outcomes or better health in general. Given that the CF population is relatively small, it is difficult to get patients and often times the experimental size is too small to provide statistically meaningful results. Participation is greatly needed to test new drugs on the horizon bringing the medical community and researchers closer to a cure.