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A new drug that suppresses the body's immune reaction for the prevention of graft rejection in heart transplant recipients has been approved by the FDA.
Prograf (tacrolimus), available in capsule form and as an injectable, was previously approved for the prevention of graft rejection in people receiving liver and kidney transplants. Prograf acts by a mechanism similar to cyclosporine, another immunosuppressant used to prevent transplant rejection. Prograf offers an alternative to cyclosporine for use in certain combination immunosuppressive regimens in liver, kidney, and heart transplantation.
"This approval is another example of the benefits of our agency's 'orphan' drugs program," says Steven Galson, M.D., director of the FDA's Center for Drug Evaluation and Research, "which seeks to answer the medical needs of small groups of patients."
In a U.S. study, patient and graft survival at 12 months after transplantation in the Prograf group was similar to that of the cyclosporine group.
The use of Prograf is associated with increased risk of neurotoxicity, renal function impairment, infection, and post-transplant diabetes mellitus. Like most combination drugs used in organ transplants, the Prograf-based combination is associated with an increased risk of malignancies, especially non-melanoma skin cancers. Prograf is manufactured by Astellas Pharma US Inc., Deerfield, Ill.
The FDA has approved the first generic capsule dosage form of zidovudine to treat HIV/AIDS to be marketed in the United States. The tablet and oral solution forms of zidovudine were previously approved for sale in the United States when the patent on those dosage forms expired in September 2005.
The approval of the capsule form of the drug, which is manufactured by Aurobindo Pharma Ltd. in Hyderabad, India, follows the expiration of GlaxoSmithKline's patent on its capsule form of the product marketed under the trade name Retrovir.
"This is a significant generic approval," says Acting Commissioner of Food and Drugs Andrew C. von Eschenbach, M.D. "Retrovir, which was initially approved in March 1987, was the first of a group of breakthrough medications that have transformed what was then a disease with a very dismal prognosis into one with a much more hopeful prognosis. Approval of this additional dosage form of zidovudine should help reduce the cost of this therapy for American patients."
Zidovudine is in the class of drugs called nucleoside reverse transcriptase inhibitors, which help keep the AIDS virus from reproducing. This anti-retroviral drug is intended to be used with other anti-retroviral agents for the treatment of HIV-1 infection.
The FDA has approved a Humanitarian Device Exemption (HDE) for Fetoscopy Instrument Sets used to treat fetuses with a rare disorder of the placenta, called twin-to-twin transfusion syndrome (TTTS), under the Humanitarian Use Device (HUD) program.
TTTS sometimes occurs when women are pregnant with identical twins. During development, blood vessels in the fetuses' shared placenta connect their blood circulations. In most cases, the blood flows properly through these vessels. In TTTS, however, the blood begins to flow unevenly, with one fetal twin receiving too much blood (the recipient) and one receiving too little (the donor). This condition can cause heart failure in the recipient twin and life-threatening anemia in the donor twin. Many of these TTTS babies do not survive delivery or are born with severe handicaps.
The Fetoscopy Instrument Sets give doctors a new option for treating TTTS, and can help prolong the mother's pregnancy and improve the odds of survival for both twins. Complications also are reduced for one or both twins.
The instrument sets are intended to be used for the treatment of TTTS for fetuses whose gestational age is between 16 weeks and 26 weeks. The sets consist of a telescopic camera used to view a fetus (fetoscope) and sheaths that are used to pass other surgical instruments and fluid through the entry site.
The HUD program encourages the development of medical devices intended to treat or diagnose a disease or condition affecting fewer than 4,000 people in the United States a year. To receive approval of an HDE application, a sponsor must demonstrate the safety and probable benefit of a device.
The FDA has approved a biologics license application (BLA) for Myozyme (alglucosidase alfa, rhGAA), the first treatment approved for people with Pompe disease, a rare but severely debilitating disease that affects between 40,000 and 300,000 Americans.
Pompe disease is inherited and caused by the deficiency or lack of the enzyme acid alpha-glucosidase, which is essential for normal muscle development and function. The disease drastically reduces a person's muscle and respiratory function, and usually results in death from respiratory failure. Pompe disease is rapidly fatal in newborn babies.
Myozyme had been granted orphan drug designation by the FDA and was approved under a priority review. Orphan products are developed to treat rare diseases or conditions that affect fewer than 200,000 people in the United States. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first sponsor who obtains marketing approval for a designated orphan drug.
The FDA approved Myozyme for administration by intravenous infusion of solution into a vein. The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion.
Patient survival without needing invasive ventilatory support was substantially greater in the Myozyme-treated infants compared with the known high mortality of untreated patients of similar age and disease severity. The drug's safety and effectiveness in other forms of Pompe disease have not been adequately studied.
A boxed warning is included in the Myozyme label to warn about the possibility of life-threatening allergic reactions. Myozyme is manufactured by Cambridge, Mass.-based Genzyme Corp.
The FDA has approved an imaging system that can help detect an indication of the possible development of cervical cancer.
The LUMA Cervical Imaging System, made by MediSpectra Inc., Lexington, Mass., can help identify sites on the cervix that may contain precancerous cells. It is intended to be used along with colposcopy, a high-magnification evaluation of the cervix, for women who have recently had an abnormal Pap test. The firm's study of the device showed that it can detect additional cancer precursors missed by colposcopy.
"Cervical cancer is one of the few highly preventable cancers," says Daniel Schultz, M.D., director of the FDA's Center for Devices and Radiological Health. "The early detection and removal of pre-cancerous cervical lesions reduces the risk of developing invasive cervical cancer."
The LUMA system shines a light on the cervix and analyzes how different areas respond to this light. The system then assigns a number, or score, to tiny areas of the cervix, and produces a color map that helps the doctor decide where to take a sample tissue, called a biopsy. The colors and patterns on the map help distinguish between healthy and potentially diseased tissue.
The FDA cautions that use of the LUMA device is not a substitute for a thorough colposcopic exam, which should be performed first to identify areas on the cervix to perform a biopsy.
In March 2006, the FDA approved the use of Relenza (zanamivir, for inhalation) for the prevention of influenza in adults and children ages 5 years and older. Relenza, an antiviral medication, was previously approved for the treatment of influenza A and B viral infections in adults and children.
The approval of Relenza for prevention gives Americans another option for preventing influenza A and B infections. Tamiflu (oseltamivir phosphate) was previously approved for both prevention and treatment of flu.
The effectiveness of Relenza in preventing seasonal influenza has been demonstrated in four large-scale studies comparing the drug with a pill containing an inactive ingredient (placebo). In two of these trials, the drug substantially reduced the spread of influenza in the participating households in which participants were 5 years or older.
In the other two trials, conducted in communities experiencing a flu outbreak, Relenza reduced the incidence of the disease in both young and older populations. In the first study, with participants ages 18 years and older, the proportion of people who developed symptoms confirmed to be flu was 6.1 percent for the placebo group and 2.0 percent for the Relenza group. The second community study enrolled people ages 12 to 94, with just over half older than 65. In this trial, the percentage of people who developed symptoms confirmed to be flu was 1.4 percent of the participants who took the placebo and 0.2 percent of those who used Relenza.
Breathing problems (bronchospasm), including deaths, were reported in some patients after the initial approval of Relenza. Most of these patients had asthma or chronic obstructive pulmonary disease. Because of these reports, Relenza is not recommended for treatment or prevention of seasonal influenza in individuals with these or other underlying airway diseases.
Relenza has not been proven effective for flu treatment in people with underlying airway disease, or for flu prevention in nursing homes. The drug also is not a substitute for the flu vaccine, which is the primary way to prevent the flu. Consumers should continue receiving an annual flu vaccination according to guidelines on immunization practices.
In preparation for a potential pandemic, the FDA has assembled the Pandemic Influenza Preparedness Task Force to provide policy leadership and strategic planning on pandemic influenza.
The FDA also is working with the pharmaceutical industry to make available appropriate products and to ensure that mechanisms are in place to collect the necessary efficacy and safety information. Both Relenza and Tamiflu have been identified for stockpiling.
Relenza is manufactured and distributed by GlaxoSmithKline Inc., based in Research Triangle Park, N.C.
The FDA has approved the first generic version of Bristol-Myers Squibb's Pravachol (pravastatin sodium) Tablets, an important step in the agency's effort to increase the availability of lower-cost generic medications.
Pravastatin is indicated for the treatment of individuals who have high cholesterol levels (hyperlipidemia) or who are at increased risk for atherosclerosis-related cardiac and cardiovascular events, such as heart attack and stroke in which high cholesterol levels are a factor. In 2005, Pravachol was the 22nd highest-selling brand-name drug in the United States, with sales totaling $1.3 billion.
"This approval is another example of our agency's endeavor to counter rising health care costs by approving safe and effective generic alternatives as soon as the law permits," says Scott Gottlieb, M.D., deputy commissioner for medical and scientific affairs. "Pravastatin is a widely-used cholesterol-lowering agent, and its generic version can bring significant savings to the millions of Americans with this disease."
Pravastatin Sodium Tablets (10 mg, 20 mg, and 40 mg) are manufactured by Teva Pharmaceutical Industries Ltd. in Kfar Sava, Israel.
Food processors and manufacturers who market foods that contain a specified amount of barley may include a health claim that their products reduce the risk of coronary heart disease, under a new FDA rule. Specifically, whole grain barley and dry milled barley products such as flakes, grits, flour, and pearled barley, which provide at least 0.75 gram of soluble fiber per serving, may bear the following claim:
"Soluble fiber from foods such as [name of food], as part of a diet low in saturated fat and cholesterol, may reduce the risk of heart disease. A serving of [name of food] supplies [x] grams of the soluble fiber necessary per day to have this effect."
Coronary heart disease claims the lives of nearly 500,000 Americans each year. High total cholesterol levels and high levels of low density lipoprotein (LDL) cholesterol are known to increase one's risk for heart disease, so consumers are encouraged to keep these levels as low as possible. Scientific evidence indicates that including barley in a healthy diet can help reduce the risk of coronary heart disease by lowering LDL and total cholesterol levels.
"FDA is pursuing new initiatives to help consumers improve the choices they have for healthy and nutritious diets," says FDA Deputy Commissioner Scott Gottlieb, M.D. "We firmly believe that one of the best ways to encourage healthier eating habits is to help consumers get truthful, up-to-date, science-based information about food products so that they can make choices that are based on a better understanding of the health consequences of their diets."
The FDA, in cooperation with the National Association of State Departments of Agriculture, the USDA's Food Safety and Inspection Service, and the U.S. Department of Homeland Security, has announced the availability of a model Food Emergency Response Plan. The goal of the response plan is to enhance the protection of the nation's agricultural industry and food security through prevention, detection, response, and recovery efforts.
The model plan provides states with a tool to use in developing their own response plan for responding to a food-related emergency.
"FDA remains vigilant in its mission to protect our country's food supply and continues to maintain collaborative partnerships with our federal and state partners by planning for, monitoring, and reacting to any potential threats," says Robert E. Brackett, Ph.D., director of the FDA's Center for Food Safety and Applied Nutrition. "By collaborating more closely with our partners involved in food safety and security, we will better leverage all of the available resources to be better prepared for any food emergency incident."
A food-related emergency involves the unintentional or deliberate contamination, threatened or actual, of food that may impact human health.
The model plan establishes a uniform structure and content that will result in response plans similar among all states. Plans developed in a similar manner will facilitate seamless regional and national responses to food emergencies.
The response plan was developed through a federal–state cooperative agreement and in consultation with a consortium of stakeholders.
The FDA has approved Dacogen (decitabine) injection for treating a condition in which a person's bone marrow does not produce enough mature blood cells.
People with myelodysplastic syndromes (MDS) have a lack of healthy blood cells that can function properly. Dacogen, manufactured by Pharmachemie B.V. Haarlem, The Netherlands, is thought to work by promoting normal development of blood cells.
The drug received orphan drug status for MDS because the condition affects fewer than 200,000 people in the United States.
MDS can develop after treatment with drugs or radiation therapy for other diseases, or it can develop without any known cause. Some forms of MDS can progress to acute myeloid leukemia, a type of cancer in which too many white blood cells are made.
The FDA's approval of Dacogen "offers patients with this rare disease an additional treatment option that may help these patients avoid blood transfusions," says Steven Galson, M.D., director of the FDA's Center for Drug Evaluation and Research.
The most common side effects reported in clinical studies of Dacogen included low white blood cell count (neutropenia), low platelets in the blood (thrombocytopenia), anemia, fatigue, fever, nausea, cough, bleeding in the skin, constipation, diarrhea, and high blood sugar (hyperglycemia).
The FDA has approved Remicade (infliximab) to treat children with active Crohn's disease, a chronic, inflammatory condition of the bowel that can be severely debilitating. Remicade was first approved in 1998 to treat Crohn's disease in adults.
Remicade is a genetically engineered monoclonal antibody that is manufactured using cells containing human and mouse antibody genes. This biotechnology product, which is taken intravenously, blocks the action of a protein called tumor necrosis factor-alpha (TNF-alpha), one of the underlying causes of inflammation in Crohn's disease.
The inflammation often involves the entire wall of the gastrointestinal tract and can result in abdominal pain, gastrointestinal bleeding, diarrhea or loose stools, blockages, and infections.
Steven Galson, M.D., director of the FDA's Center for Drug Evaluation and Research, notes that there have been no satisfactory treatments for children with moderately to severely active Crohn's disease who have symptoms despite conventional therapies.
"Remicade is not a cure, but it provides a much-needed option for reducing the symptoms and inducing and maintaining disease remission in children who have no other safe and effective therapy," says Galson. "We believe that the potential benefits of this product outweigh the risks that are known and have been carefully evaluated."
The safety profile for Remicade in the children's study was similar to the data that were presented at an FDA Arthritis Advisory Committee meeting in March 2003, and that dealt with the extent to which anti-TNF therapies may increase the risk of serious infections and illnesses, such as sepsis and pneumonia, in certain people.
These risks, which are described in a study in the May 17, 2006, issue of the Journal of the American Medical Association, are included in the current labels for all approved TNF-alpha blocking agents, including Remicade. More recently, the FDA has received rare reports of an aggressive and often fatal type of cancer (hepatosplenic T-cell lymphoma) in adolescents and young adults with Crohn's disease who were treated with Remicade. Most of these people were receiving standard immunosuppressive therapies, such as azathioprine or 6-mercaptopurine, along with Remicade. The FDA is working with the manufacturer, Centocor Inc. of Malvern, Pa., to address this risk by updating the "warnings" sections of the Remicade label.
The FDA continues to monitor the safety of Remicade and similar treatments to maximize their benefits yet limit, to the degree possible, the potential for very serious side effects.
The FDA has approved an application to resume the marketing of Tysabri (natalizumab) under a special restricted distribution program. Tysabri is a drug used to treat people with relapsing forms of multiple sclerosis (MS) to reduce the frequency of flare-ups.
Tysabri is approved for use as a monotherapy, meaning it should not be used with other drugs that modify the immune system. Tysabri, which is injected directly into a vein (infused), is for people who have not responded to, or cannot tolerate, other treatments for MS.
The FDA initially approved Tysabri in November 2004, but the manufacturer Biogen Idec Inc. withdrew the drug in February 2005 after three patients in clinical trials developed a serious and rare viral infection of the brain. Two of the cases were fatal. On the basis of this information, the FDA put clinical trials of the drug on hold in February 2005. The FDA allowed a clinical trial of Tysabri to resume in February 2006 after reexamining the patients who had participated in previous clinical trials and confirming that there were no additional cases of PML.
To decrease the possibility of people developing PML in the future, while also making Tysabri available to those with MS who could benefit from it, the FDA consulted in March 2006 with its Peripheral and Central Nervous System Drugs Advisory Committee. The committee recommended a risk-minimization program with mandatory patient registration and follow-up to identify as early as possible any cases of PML that may occur and why. In response, Biogen Idec submitted to the FDA a risk management plan, called the TOUCH Prescribing Program, to help ensure safe use of the product.
After a thorough review of the information provided by Biogen Idec, the FDA determined that Tysabri can be made available under the TOUCH Program. Biogen Idec and its distributor for Tysabri, Elan Pharmaceuticals Inc., will manage the program, which includes the following main features:
The FDA has approved Azilect (rasagiline), a new drug for the treatment of Parkinson's disease. Azilect is a monoamine oxidase type B (MAO-B) inhibitor that blocks the breakdown of dopamine, a chemical that sends information to the parts of the brain that control movement and coordination.
"This is a welcome development for the more than 50,000 Americans who are each year diagnosed with Parkinson's disease," says Steven Galson, M.D., director of the FDA's Center for Drug Evaluation and Research. "Parkinson's is a relentless disease with limited treatment options, and each new therapy is an important addition to the physicians' treatment options."
Parkinson's disease is a chronic, progressive neurodegenerative condition caused by the destruction of the brain cells that produce dopamine. As the level of this chemical declines, messages from the brain telling the body how and when to move are delivered more slowly, leaving a person incapable of initiating and controlling movements in a normal way.
Azilect was approved for use as an initial single drug therapy in early Parkinson's disease, and as an addition to a standard Parkinson's disease treatment, levodopa, in people with more advanced disease.
People who take Azilect may have a sudden, large increase in blood pressure (hypertensive crisis) if they also consume tyramine-rich foods or beverages (such as cheese and red wine) or dietary supplements or amines contained in many cough and cold medications. A hypertensive crisis can lead to a stroke and death. People taking Azilect will need to avoid sources of tyramines and amines. Like most other medications for Parkinson's, Azilect has the potential to cause involuntary movements, hallucinations, and lowered blood pressure.
During Azilect's development, melanoma was diagnosed in a small number of people treated with the drug. The manufacturer, Teva Pharmaceutical Industries Ltd., Israel, will perform an after-market study to determine whether or not Azilect increases the risk of this form of skin cancer. The product labeling will recommend that people who take Azilect get periodic dermatologic examinations.