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FDA
FDA Consumer
magazine
July-August 2003 Issue
Topics in This Issue:
Doctors now have another tool to evaluate the response of their asthma patients to treatment with anti-inflammatory drugs. In May, the FDA cleared the NIOX Nitric Oxide Test System, a first-of-a-kind non-invasive system that measures the concentration of nitric oxide in exhaled human breath. A decrease in exhaled nitric oxide concentration suggests that anti-inflammatory treatment may be decreasing asthma-related lung inflammation. The lungs of people with asthma become inflamed and constrict, limiting airflow and making it hard to breathe. The disease affects about 15 million Americans.
With the NIOX system, patients use a mouthpiece that is connected to a computer by a breathing tube. The patient inhales nitric oxide-free air to total lung capacity and then exhales slowly into the mouthpiece. The nitric oxide concentration is displayed immediately on the computer screen.
The FDA cleared the NIOX system based on clinical studies of 65 patients--adults and children ages 4 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. Results showed that most patients had a 30 percent to 70 percent decrease of nitric oxide levels after two weeks of treatment with inhaled steroids.
The NIOX system is made by Aerocrine AB of Stockholm, Sweden.
The FDA is urging health care providers and people who take the cholesterol-lowering drug Lipitor (atorvastatin) to examine labels on the drug carefully following reports of counterfeit versions of the drug.
In May, Albers Medical Distributors Inc. recalled three lots of 90-count bottles of Lipitor. The company warned that these lots represent a potentially significant risk to consumers. The product was repackaged by Med-Pro Inc. of Lexington, Neb.
The lots in that recall were:
In June, the FDA announced that its investigation had uncovered additional counterfeit Lipitor:
Anyone who has Lipitor with these lot numbers should not take it and should return the product to the pharmacy where it was purchased. Many people who take Lipitor do not receive it in the 90-tablet bottles being recalled, but in smaller quantities from their pharmacists. People taking Lipitor who have questions about their product should check with their pharmacists. The FDA is working closely with Pfizer Inc., the manufacturer of Lipitor, as well as with health professionals on this counterfeit problem.
In carrying out its public health mission, the FDA regularly conducts investigations and testing to identify and remove products from the market that are counterfeit, have been tampered with, or are otherwise unsuitable. The FDA's Office of Criminal Investigations (OCI) continues to investigate the matter.
In April, the Pharmaceutical Research and Manufacturers of America, a trade association representing pharmaceutical and biotechnology companies, announced that its members would notify the FDA of suspected counterfeit cases within five working days as part of a voluntary program to combat the practice.
Chemotherapy is often distressing for cancer patients due to the debilitating nausea and vomiting associated with the treatment. The symptoms can even cause patients to refuse further chemotherapy treatments. A new drug, recently approved by the FDA, can help prevent these symptoms when used in combination with other anti-nausea and anti-vomiting drugs.
Emend (aprepitant) is the first approved therapy that prevents the nausea and vomiting that many patients experience more than 24 hours after receiving chemotherapy.
Emend is given along with two other drugs for three days, starting just before a chemotherapy treatment is administered. It reduces nausea and vomiting in a new way--by blocking brain receptors called NK1 receptors. Emend can reduce nausea associated with chemotherapy treatments for cancers such as lung cancer, head and neck cancer, and some female cancers.
Over 1,000 people with cancer were studied while taking Emend, manufactured by Merck & Co. Inc., of Whitehouse Station, N.J. Fewer patients had symptoms of nausea and vomiting when the new drug was part of their treatment, compared with patients receiving standard medicines.
The FDA has approved Fabrazyme (agalsidase beta), the first treatment for people with Fabry's disease. This serious genetic metabolic disorder affects about 1 in 40,000 men. Though it is believed that fewer women suffer the most serious consequences of the disease, they can also be seriously affected.
Because of a deficiency in the enzyme alpha-galactosidase A, Fabry's disease causes certain fats to accumulate in the blood vessels over many years, damaging various tissues and organs such as the kidneys and heart. As a result, people with Fabry's disease often must cope with significant pain and disability, and they typically have a shortened life span.
Fabrazyme is a version of the human form of the natural enzyme produced by recombinant DNA technology. When given intravenously, this replacement of the missing enzyme reduces a particular type of fat accumulation in many types of cells, including blood vessels in the kidneys and other organs. It's believed that this reduction of fat deposition will prevent the development of life-threatening organ damage.
The FDA approved Fabrazyme in April under an early approval mechanism. This policy accelerates approval for therapies that treat serious or life-threatening illnesses when studies indicate that early favorable outcomes are likely to predict clinical benefit. In this case, the manufacturer of Fabrazyme, Genzyme Corp. of Cambridge, Mass., performed biopsies looking at the cells lining the blood vessels within the kidneys and other organs in people with Fabry's disease. Many of the cells examined have shown significant clearance of fat deposits. Genzyme has committed to continue its clinical trial to verify Fabrazyme's benefit, and has set up a voluntary patient registry to follow the long-term progress of people treated with Fabrazyme.
More than half of all new kidney transplant patients could potentially benefit from a newly approved drug regimen. The FDA has revised the labeling for Rapamune (sirolimus)--an anti-rejection medication--to allow new kidney transplant patients at low to moderate risk of organ rejection to stop taking cyclosporine two to four months after transplantation. By substituting higher levels of Rapamune for cyclosporine, it is hoped that kidney function will improve.
Currently, all kidney transplant patients are treated with a combination of medications that "turn off," or suppress, the body's immune response so that it will not reject the new organ. Three or more drugs, such as cyclosporine and steroids, are typically used. The revised labeling for Rapamune is the first approval of a cyclosporine-sparing regimen for new kidney transplant patients.
The combined use of Rapamune and cyclosporine may carry long-term risks to the transplanted kidney's function, but the newly approved regimen using higher levels of Rapamune may help kidney transplant patients get off cyclosporine sooner without increased risk of organ rejection. Stopping the use of cyclosporine earlier is likely to be associated with improved kidney function.
In 2000, the year the most recent statistics are available, there were more than 14,000 kidney transplants in the United States, according to the National Institute of Diabetes and Digestive and Kidney Diseases.
Wyeth-Ayerst Pharmaceuticals Inc. of Philadelphia is the sponsor of the approved new drug application for Rapamune.
Acromegaly, a potentially life-threatening disease triggered by an excess of growth hormone, causes headaches, profuse sweating, swelling, joint disorders, changes in facial features, and enlarged hands, feet, and jaw. If untreated, people with acromegaly often have a shortened life span because of heart and respiratory diseases, diabetes, and cancer.
The FDA recently approved Somavert (pegvisomant) to treat the condition. The drug is the first in a new class of drugs called growth hormone receptor antagonists. Somavert is approved for people who have not responded adequately to existing therapies. The most commonly reported side effects with Somavert in clinical trials were injection site reactions, sweating, headache, and fatigue. People should have tests to monitor liver function during the first six months of therapy.
Somavert will be marketed by Pfizer Inc. of New York City.
A recently established partnership will bolster the FDA's ability to use the best science and the latest technology available to improve risk management of marketed products.
The FDA has established a cooperative research and development agreement (CRADA) with Lincoln Technologies Inc. of Wellesley Hills, Mass. The purpose of this partnership is to use "data mining" to enhance the FDA's ability to monitor the safety of drugs, biologics and vaccines after they have been approved for use.
Data mining is a technique using modern computing and statistical algorithms to extract meaningful, organized information from large, complex databases. Under the CRADA, the technique will be applied to data that the FDA already collects about adverse events involving approved drugs, biologics and vaccines. The specific data set used for data mining purposes does not include patient names, addresses, Social Security numbers or similar information.
Data collected from millions of suspected drug-related adverse event reports and other electronic medical information could aid the FDA in identifying "signals" of adverse events and the patterns in which they occur. With more effective tools to detect such patterns, the FDA can act faster to evaluate and respond to these reports. For example, effective data mining would allow the agency to identify a pattern of adverse drug events in a specific population, and the agency could then communicate this knowledge sooner to medical professionals and patients.
People who experience the sustained, simultaneous muscle contractions of the rare movement disorder called dystonia now have another treatment for the condition, which forces affected body parts into abnormal and sometimes painful postures and movements.
The Activa Dystonia Therapy System, a brain implant already used to treat Parkinson's disease and essential tremor, was approved by the FDA in April to treat dystonia. The deep brain stimulator is manufactured by Medtronic Inc. of Minneapolis.
The system consists of electrodes and a neurostimulator. The electrodes are implanted into the brain and connected by wires under the skin to a neurostimulator implanted in the chest. The neurostimulator sends a constant stream of tiny electrical pulses to the brain, which suppresses symptoms. When the device is implanted in both sides of the brain, two separate systems are used. Patients touch a hand-held magnet over the neurostimulator to switch the device on and off.
The most recent approval broadens the use of the device to people with primary dystonia who get little or no relief with medication. Primary dystonia has no known cause, unlike secondary dystonia, which is caused by an underlying disease such as Parkinson's, a brain tumor, or a stroke. Results in patients using the device varied, and the specific benefit for an individual cannot be predicted. But because of the debilitating nature of dystonia and the lack of other effective treatments, the FDA has determined that the probable health benefit for people with primary dystonia who get no relief with medication outweighs the risk of illness or injury from the device.
The FDA approved the device through a special regulatory process known as a humanitarian device exemption, which is designed to encourage the development and marketing of medical devices for people with rare conditions.
The first treatment for people with certain forms of a rare genetic disease called MPS I has been approved by the FDA. MPS I occurs when a particular enzyme is absent or malfunctions in the body. The enzyme normally breaks down molecules called glycosaminoglycans (GAG) in the cells. People with MPS I have a buildup of GAG in the cells, resulting in progressive cellular damage that affects appearance, physical abilities, organ functions and, in some cases, mental development.
The new biotechnology product, Aldurazyme (laronidase), is a version of the deficient enzyme. Aldurazyme helps prevent the buildup of GAG in the cells and has been shown to improve lung function and exercise ability.
Aldurazyme is approved for people with the Hurler and Hurler-Scheie forms of MPS I as well as those with the Scheie form with moderate to severe symptoms. Hurler syndrome is the most severe of the MPS I forms. Children with Hurler syndrome often die early from respiratory diseases and cardiac complications. Hurler-Scheie syndrome is less severe, but people who have it usually do not survive beyond their early 20s. Scheie syndrome is the mildest form, with many patients living well into adulthood.
Studies in Canada indicate that 1 in 100,000 babies born has Hurler syndrome, 1 in 115,000 has Hurler-Scheie syndrome, and 1 in 500,000 has Scheie syndrome. Similar studies have not been done in the United States.
Aldurazyme is manufactured by BioMarin Pharmaceutical Inc. of Novato, Calif.
The first stent containing a drug that prevents coronary arteries from becoming clogged again after angioplasty has been approved by the FDA. So-called drug-eluting stents that combine drugs with medical devices may have a substantial impact on the occurrence of reblockages in patients who have heart disease.
The Cypher Sirolimus-Eluting Coronary Stent, a tiny metal mesh tube covered with the drug sirolimus, provides a mechanical scaffold to keep an artery open, while the drug is slowly released from the stent to prevent the build-up of new tissue that can reclog arteries.
Each year, 800,000 angioplasty procedures are performed in the United States to open clogged coronary arteries. In about 15 percent to 30 percent of patients, the artery becomes clogged again (restenosis) within a year, and it must be treated again with a procedure such as angioplasty or bypass surgery. In studies conducted by the manufacturer, Cordis Corp. of Miami Lakes, Fla., the stent reduced the rate of restenosis by about two-thirds.
Patients who are allergic to sirolimus or to stainless steel should not receive a Cypher stent. The FDA also cautions those who have had recent cardiac surgery and women who may be pregnant or are nursing. Patients who receive the drug-eluting stent likely will be required to take certain kinds of anti-platelet drugs for at least several months.
The FDA is requiring Cordis to conduct a 2,000-patient post-approval study to assess the long-term safety and effectiveness of the Cypher stent and to look for rare adverse events that may result from using the product.
Gleevec (imatinib mesylate) tablets were approved in May to treat children with Philadelphia chromosome positive chronic myeloid leukemia (CML)--a rare, life-threatening form of cancer that accounts for 2 percent of all leukemia in children. This marks the first approval of a new pediatric cancer drug in more than a decade.
Gleevec is indicated for children whose disease is in the chronic phase and has recurred after stem cell transplant or for those who are resistant to interferon alpha therapy.
The FDA originally approved Gleevec in 2001 for CML in adults. It was approved to treat gastrointestinal stromal tumors in February 2002.
"We hope to see more products developed that improve pediatric cancer care, and we are working to facilitate their development and timely approval," FDA Commissioner Mark B. McClellan, M.D., Ph.D., said in announcing the pediatric approval.
The new treatment was evaluated under the FDA's accelerated approval program. Gleevec was cleared for use in children based on data from its use in adults with CML, combined with study results showing good responses in a small number of children. More studies will be done to confirm that the drug has improved survival or resulted in other clinical benefit for children.
The most frequently reported problems with the use of Gleevec are nausea, vomiting, diarrhea, swelling that can be severe (edema), and muscle cramps. A considerable reduction in white blood cells and platelets also was reported with Gleevec treatment. Gleevec can be given to children once a day, or the daily dose may be split in two--once in the morning and once in the evening.
Gleevec is manufactured by Novartis Pharma AG for Novartis Pharmaceuticals Corp. of East Hanover, N.J.
A new test will allow doctors to diagnose a potentially life-threatening fungal infection sooner. Invasive aspergillosis infections occur in people with leukemia, people who have received organ and bone marrow transplants, and in people whose immune systems are compromised by illness or chemotherapy. Although the number of invasive aspergillosis cases is estimated to be only a few thousand per year, the disease is very serious and has a mortality rate of between 50 percent and 100 percent.
The FDA cleared the test, Platelia Aspergillus EIA, in May. The test detects Aspergillus galactomannan antigen in blood, and results are available in about three hours. By comparison, it takes at least four weeks before results are available with the standard culture method of testing for Aspergillus.
Clinical studies at three cancer centers showed the test could accurately identify the presence or absence of the Aspergillus antigen. The centers tested 1,890 blood samples collected from 170 people. Thirty-one patients had proven or probable cases of invasive aspergillosis. The new test correctly identified 25 of the 31 people who had Aspergillus antigen. When 148 people without symptoms were tested, the test correctly identified 132 of the 148 as not having the antigen.
The Platelia Aspergillus EIA test is manufactured by Bio-Rad Laboratories of Hercules, Calif.
A permanently implanted device called Enteryx has been approved by the FDA to help people with the symptoms of gastroesophageal reflux disease (GERD). GERD is a condition in which some of the stomach's contents--including acid--backs up (refluxes) into the esophagus, causing heartburn or burning pain in the chest or back of the throat. More than 60 million American adults experience GERD, and about 25 million of them have daily symptoms.
Inserted through a thin tube called an endoscope, the device prevents the reflux of stomach acid into the throat, potentially allowing people with chronic GERD to avoid taking medications daily.
Mild heartburn can be treated by adopting dietary changes such as avoiding foods that cause heartburn and eating smaller portions. Treatment of chronic GERD, however, may also require prescription drugs to help keep the acid secretion in the stomach at a reduced level.
Enteryx, a product of Enteric Medical Technologies of Foster City, Calif., a wholly-owned subsidiary of Boston Scientific Inc., is approved for use in people who have GERD symptoms and who require and respond to certain medications. The device, a solution made up of a polymer and a solvent, is injected during an X-ray guided procedure into the wall of the lower esophagus. After the injection, the solvent separates away and the polymer solidifies into a spongy material that is intended to help prevent the reflux.
The device has been found to eliminate or reduce the need for medications and to improve the symptoms of GERD. In a year-long study of 85 patients, about two-thirds were able to discontinue all of their medications, while a few (9 percent) could reduce their dosage by at least half. Most (72 percent) noted an improvement in their symptoms when compared with taking no medications prior to the implant.
Although most of those in the study had improvements in their symptoms and medication requirements, evaluations of the esophagus done during the clinical trial showed evidence of persistent acid reflux in 61 percent of participants and low-grade inflammation in 37 percent of participants at 12 months.
The most common side effect seen with the Enteryx treatment was pain beneath the breastbone that usually lessened within two weeks. Other common side effects included temporary difficulty with swallowing, fever, sore throat, and gas, bloating, and belching.
The device should not be used in people who are unable to undergo endoscopy, or who have dilated veins in the esophagus due to liver disease.
Efforts to ensure that the food Americans eat is safe are working, according to a report titled "Scientific Criteria to Ensure Safe Food," released in April by the National Academy of Sciences. The report, sponsored by the FDA and the U.S. Department of Agriculture, reinforces that significant progress has been made in reducing and preventing foodborne illness.
The report specifically cited the adoption of the Hazard Analysis and Critical Control Point (HACCP) approach to food safety, which the FDA requires for seafood and fresh juice. The HACCP approach is voluntary in the dairy industry, which has shown some progress in improving the safety of its products.
The FDA has announced labeling changes for Zocor (simvastatin), which the agency approved in 1991 to lower cholesterol. The new labeling reflects results of the Heart Protection Study (HPS), a clinical trial involving more than 20,500 people. This research showed that Zocor is effective in reducing risks of fatal and non-fatal heart attacks and strokes, and in reducing the need for bypass surgery and angioplasty.
In the study, men and women who have heart disease or are at high risk because of diabetes, peripheral arterial disease, or a history of stroke or other cerebrovascular disease, were treated with either Zocor or a placebo for an average of five years. The average age of patients entering the HPS was 64 and the average LDL-C (low density lipoprotein C or "bad" cholesterol) level at baseline was 131 milligrams per deciliter. The trial population included a large number of people with diabetes and older people.
The risk of death from coronary heart disease was reduced by 18 percent in those treated with Zocor. The risk of having a non-fatal heart attack was reduced by 38 percent in this group. Zocor also reduced the risk of stroke by 25 percent and reduced the need for undergoing procedures to unblock clogged arteries.
The effect of Zocor in reducing the rate of cardiovascular events was seen in a number of subpopulations of people enrolled in the trial, including those with and without heart disease or diabetes, and regardless of gender, age, or baseline cholesterol levels. An important observation was that people who had diabetes, peripheral vessel disease, and cerebrovascular disease, but who had no evidence of heart disease, benefited from taking Zocor. The drug has been shown to be effective in reducing total cholesterol and LDL cholesterol.
Zocor is one of a class of cholesterol-lowering drugs called statins. As with other statins, Zocor should be used with a standard cholesterol-lowering diet. The dose of Zocor should be determined according to the goals of therapy and the response to the drug. People taking this type of medication should also be aware of any muscle pain, which may indicate rhabdomyolysis, a muscle breakdown disorder. Symptoms can include fatigue, weakness, fever, nausea and vomiting, and severe muscle pain. The disorder can cause electrolyte imbalances that could result in heart rhythm problems, cardiac arrest, or heart attack.
Zocor is manufactured by Merck and Co. Inc. of Whitehouse Station, N.J.