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New FDA approvals
These drugs recently received final approval from the U.S. Food and Drug Administration (FDA):
• Agalsidase beta (Fabrazyme) by Genzyme Corp. The FDA has approved the first treatment for patients with Fabry disease.
The new product, agalsidase beta (Fabrazyme), was produced by recombinant DNA technology to be a synthetic version of a natural human enzyme. It is given intravenously. This replacement of the missing enzyme caused by Fabry disease, alpha-galactosidase A, reduces a particular type of lipid accumulation in many types of cells, including blood vessels in the kidney and other organs. It is believed likely that this reduction of fat deposition will prevent the development of life-threatening organ damage and will have a positive health effect on patients.
Agalsidase beta was approved under an accelerated approval mechanism through the FDA’s orphan drugs program. One of the requirements of an accelerated approval is that the sponsor completes a postmarket study verifying that patients will benefit from the product. Genzyme says it will continue conducting an ongoing randomized placebo-controlled trial to verify agalsidase beta’s benefit to patients and to assess the drug’s effects on the progression of kidney and heart disease and the incidence of strokes.
In addition, Genzyme has set up a patient registry to follow the long-term progress of patients. Enrollment in this registry is voluntary.
In clinical studies of agalsidase beta, the main safety concern in patients receiving the drug was infusion reactions, some of which were severe. These include fever, chest tightness, blood pressure changes, abdominal pain, and headache. Most patients also develop antibodies to the product, and some patients who experience allergic reactions may need to be further evaluated. Because of the potential for these severe reactions, appropriate medical observation and support should be available when agalsidase beta is administered.
• The NIOX Nitric Oxide Test System by Aerocrine AB, of Sweden. The FDA has cleared for marketing a first-of-a-kind noninvasive test system to measure the concentration of nitric oxide in exhaled human breath. The test system should help make it easier for doctors to monitor a patient’s asthma.
The test system combines equipment that detects nitric oxide and equipment that analyzes exhaled breath with a special computer system. To use this new device, patients place a mouthpiece, connected by a breathing tube to the computer, over their mouth. They inhale nitric oxide-free air to total lung capacity, then slowly exhale into the mouthpiece. The nitric oxide concentration is displayed immediately on the computer screen.
The FDA cleared the NIOX system based on clinical studies conducted by the manufacturer of 65 patients, both adults and children ages four years and older, with confirmed diagnoses of asthma. The patients were tested with the NIOX system before they began drug treatment and again two weeks later. The studies were conducted at nine medical centers in the United States. The results showed that most patients had a 30-70% decrease of nitric oxide levels after two weeks of treatment with inhaled steroids. In this study, elevated nitric oxide levels above 30 parts per billion correlated with moderate-to-severe asthma.