The Food and Drug Administration announced approval Thursday of a genetically engineered drug offering the first new treatment for cystic fibrosis in 30 years.
The drug promises hope for victims of an inherited disease affecting about 30,000 Americans and often leading to their deaths before age 30. Cystic fibrosis is the most common lethal inherited disease among whites. There is no cure for it.The FDA said it has licensed the production of the drug dornase alfa, called DNase, a product of recombinant DNA technology - genetic engineering.
"Although this new product is not a cure for cystic fibrosis, the clinical data show that it can make a real difference in the quality of life for many patients, FDA Commissioner David Kessler said.
"This is the first treatment that specifically improves lung function in cystic fibrosis patients," he said.
DNase is manufactured by Genentech Inc. of San Francisco and will be marketed under the trade name Pulmozyme.
Approval came following a six-month clinical trial of the drug involving 968 people suffering from cystic fibrosis. Some were given a neutral placebo and some were given the drug.
"Daily doses of DNase, when used in conjunction with standard therapies, reduced the risk of severe respiratory tract infections by 27 percent and increased patients' lung function," the FDA said.
But there were side effects: throat inflammation, chest pain, voice alteration and laryngitis.
No tests were conducted to test the drug's safety and effectiveness in children under 5 or in patients with less than a 40 percent breathing function. Nor was the drug tested to see if it would be safe and effective for more than a year.
Genentech said the drug reduces lung infections by breaking up the thick mucus accumulation that is the major complication of cystic fibrosis. There is no cure for it.
Cystic fibrosis is caused when a baby inherits a flawed gene from each parent. The disorder causes the buildup of dense mucus in the walls of the breathing airway and in the digestive tract. Bacterial infections and eventual damage to lung tissue usually result.
DNase dissolves the tangle of matter from dead cells that accumulates in the lungs. It is inhaled through a device that converts the liquid drug into an atomized spray.
The drug thins and then liquefies the mucus, which then can be coughed up.