March 29, 2024
BANGOR DAILY NEWS (BANGOR, MAINE

FDA OKs drug for cystic fibrosis> Genetically engineered medication improves quality of life

WASHINGTON — People suffering from cystic fibrosis, an inherited lung disorder that affects about 30,000 Americans, will soon have access to a new, genetically engineered drug treatment, the Food and Drug Administration announced Thursday.

The FDA said it has approved use of the drug dornase alfa, the first new treatment for cystic fibrosis symptoms in 30 years.

“Although this 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,” said FDA Commissioner David Kessler. “This is the first treatment that specifically improves lung function in cystic fibrosis patients.”

The drug, manufactured by San Francisco-based Genentech Inc., will be marketed under the trade name Pulmozyme. When used in conjunction with standard therapies in a six-month clinical trial, the drug reduced the risk of severe respiratory tract infections in 27 percent of the patients who participated and improved their lung functions.

“It is a very important milestone,” said Dr. Robert J. Beall, an executive vice president of the Cystic Fibrosis Foundation. “If we can reduce the infections, we should be able to reduce the lung destruction and if we reduce the lung destruction that ought to contribute to these patients increased survival.”

There is yet no scientific data, however, to prove that the drug could extend patient’s lives. The median survival age is now 29 years. Twenty-five years ago, it was 5 years.

The treatment does have side effects, including inflammation of the throat, chest pain, voice alteration and laryngitis, but similar effects were also noted in patients taking a placebo instead of the drug.

The drug is not for all people with cystic fibrosis. There was no attempt to test the drug’s safety and effectiveness in children under 5 or in patients with less than 40 percent breathing function. The drug was not tested to see if it would be safe and effective for more than a year.

When people have cystic fibrosis, thick mucous fills their lungs and airways, reducing their lung functions and triggering chronic lung infections. Respiratory complications are the major cause of death among patients.

Pulmozyme works by liquefying the thick mucous, making it easier to dislodge.

The FDA has approved several genetically engineered drugs over the last several years, including Interferon, to treat hepatitis and leukemia; G-CSF, to produce white blood cells for cancer patients receiving chemotherapy; GMCSF, to produce white cells for patients receiving bone marrow transplants; and Erythropoetin, to produce red blood cells for patients with kidney problems.

“We have approved a handful of drugs that are genetically engineered to treat conditions,” Dr. Jane Woodcock, a physician at the FDA. “These proteins could not be administered any other way, so we really have a new kind of drug treatment. It is hoped that this is the promise of biotechnology.


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