The U.S. Food and Drug Administration (USFDA) announced on Thursday that it has approved the use of a new drug combination designed to fight the most common form of cystic fibrosis, a genetic disease that causes the buildup of mucus within the body and results in lung infections and even death.
The new medication, called Orkambi, was developed by Vertex Pharmaceuticals Inc. to halt the effects of a variation of cystic fibrosis that currently afflicts 8,500 Americans, most of which are 12 years old and beyond.
Orkambi is a follow-up to Vertex's own groundbreaking pill, Kalydeco, which was the first of its kind to cure the primary cause of cystic fibrosis back in 2012. The new treatment is a combination of Kalydeco and a newly developed drug ingredient called lumacaftor.
"The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis," Dr. John Jenkins, director at the Center for Drug Evaluation and Research, said.
"Today's approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis."
According to Vertex, cost for the Orkambi treatment is valued at $259,000 every year, which is $311,000 more affordable than the annual cost for Kalydeco treatment.
An estimated 30,000 people living in the United States suffer from cystic fibrosis. The condition is caused by different genetic mutations that are transferred from parent to child. It causes the formation of sticky mucus in the lungs and other organs of the patient, leading to digestive problems, infections and early death.
Dr. Robert Giusti of the Langone Medical Center at New York University explained that around half of all cystic fibrosis patients in the U.S. have the type of the disease targeted by Orkambi. This particular form occurs when a child takes on two genetic mutation copies-one from each of the parent.
Giusti added that he expects that the USFDA will expand the approval of Orkambi to cystic fibrosis patients as young as six years old in order to increase the number of people who could benefit from the new drug.
"This is really exciting because this is a disease that causes one to two percent deterioration each year in lung function of patients," Giusti said.
"Now they have a therapy available to potentially reverse that effect."
Photo: Images Money | Flickr
