According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the first cystic fibrosis drug on the market that treats the underlying cause of cystic fibrosis. ORKAMBI is effective for patients that have the most common form of the disease, and had previously been approved for patients age six and older. The new approval clears the drug for use in younger patients aged 2-5 years.
About Cystic Fibrosis
Cystic fibrosis is a genetic disorder that has wide ranging systemic effects, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus provides a fertile habitat for bacteria and makes the patient highly vulnerable to lung infections. Most patients must take antibiotics for most of their lives. The disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include breathing problems, coughing up mucus, male infertility, clubbed digits, lung and sinus infections, fatty stool, and poor growth. Cystic fibrosis is a progressive disease that causes lung function to worsen over time. Treatment involves antibiotics, other medications, and, in severe cases, lung transplantation. To learn more about cystic fibrosis, click here.
Earlier Treatment, Better Outcomes
The new approval means that cystic fibrosis patients can begin treatment with ORKAMBI earlier than before, which will make it more effective and should improve overall outcomes for patients. The approval comes after the conclusion of a recent study that found that the safety profile of the drug was more or less the same in younger patients when compared to those age six and up.
About ORKAMBI
Vertex has also submitted a Marketing Authorisation Application to the European Medicines Agency (EMA) that would also expand ORKAMBI’s indication to the 2-5 year age range. A decision on this application is expected to appear early next year.
ORKAMBI was first approved by the FDA in July 2015, and marked a major milestone for treating cystic fibrosis. It is the only drug on the market that helps treat the underlying cause of the disease, but the drug has sparked some controversy, with critics saying that its limited effectiveness does not justify its extreme price.
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