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T99-3 Print Media: 301-827-6242
January 15, 1999
Consumer Inquiries: 888-INFO-FDA
To the people who struggle for survival, freedom from pain and a better quality of life, the benefits of last year's new major products are very important. This is particularly true because the 1998 approvals include a high number of monoclonal antibodies that represent significant medical advances. Indications for these products that were developed and produced through the growing field of biotechnology include breast cancer, organ rejection, Crohn's disease, and treatment of lower respiratory tract disease in children.
The following are some of the groups of patients who can benefit from the new products approved in 1998 by FDA's Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER) and Center for Devices and Radiological Health (CDRH) :
People with Cancer. Relief from cancer, the second deadliest disease in the United States, and one that affects eight million Americans, was a prime object of several major products approved last year. One new treatment that can potentially ease the suffering of many cancer patients is fentanyl citrate. It is a drug especially formulated to ease its administration to patients who are suffering from the agonizing pain that breaks through narcotic therapy.
Breast cancer victims can be treated with several new drugs approved last year.
Trastuzumab, one of the new breast cancer treatments, is an outstanding example of monoclonal antibodies bioengineered by grafting a mouse antibody onto a human antibody framework. Trastuzumab was licensed by CBER to treat patients with metastatic disease resistant to other therapies. The medicine binds to the protein HER2, a substance overexpressed by tumors in about 30 percent of the 1.6 million American women with breast cancer.
The second new medication is capecitabine, an oral treatment which is converted by the body to 5-fluorouracil (5FU), a drug that until now had to be administered intravenously. The product is used to treat patients with advanced breast cancer that does not respond to other medications.
The third new approval is a new indication for tamoxifen citrate, a well-established breast cancer drug. The product, which has been in use for more than 20 years, now is approved for the reduction of breast cancer risk in women who have a great likelihood to get the disease.
In addition to the breast cancer products, CDER approved valrubicin, a new treatment for patients with cancer of the bladder which cannot be immediately removed, and thyrotropin alfa, a new diagnostic tool for patients with thyroid cancer.
CBER also approved new cancer-related indications for three biological drugs: aldesleukin for use in metastatic melanoma; filgrastim for use in acute myeloid leukemia, and BCG live for treatment of certain tumors of the bladder.
Devices approved by CDRH added two important new tools to the oncologic armamentarium. One of them uses artificial intelligence, computer science and radiology to help screen and analyze mammograms. The apparatus highlights abnormalities that otherwise could escape the radiologist's attention.
The other new device made available last year detects free Prostate Specific Antigen, a substance associated with prostate cancer that strikes 300,000 men in the U.S. a year. Used in combination with other tests, the device can help distinguish prostate cancer from benign conditions, thereby sparing the patients unnecessary biopsies.
People with Arthritis More than two million men, women and children in the United States suffer from rheumatoid arthritis and 16 million others suffer from osteoarthritis. Three new products approved last year are designed to treat these patients.
One of the new products -- etanercept -- is an example of the way biotechnology is fulfilling its promise. A genetically engineered protein, etanercept can help to reduce the symptoms of moderate to severe rheumatoid arthritis in the estimated 1 million - 1.5 million patients whose pain has not responded to other treatments.
The other new treatment, also for rheumatoid arthritis, is leflunomide, the first oral drug that can relieve the symptoms and slow the progression of this potentially disabling autoimmune disease.
Celecoxib, the third new drug in this category, is a new type of non-steroidal anti-inflammatory pain reliever (NSAID) that can be used for the treatment of both rheumatoid arthritis and osteoarthritis. It offers an alternative therapy for both diseases.
People with Diabetes According to the Centers for Disease Control and Prevention, about 16 million Americans have diabetes, but not all of them are aware of their condition. Those whose disease is treated can be helped by three new products made available last year.
Two of them are new forms of glucagon, which is now produced by recombinant technology. As a result, the availability of this drug for patients with hyperglycemia no longer depends on an adequate supply of bovine pancreas.
The third new product is an alternative for the thousands of diabetic adults and children who must draw blood once or twice a day from their fingers. The blood sample is used for glucose analysis, which is essential for the control of the disease, but pinpricking by a lancet in time can become very trying, especially for young children. A portable, battery operated device approved by CDRH uses a laser to prick fingers in a procedure that patients describe as easier and more comfortable than the use of the lancet.
People with Hepatitis About 4 million people in this country have chronic hepatitis C infection that can lead to cirrhosis, liver cancer and liver failure, and is the most common reason for liver transplantation. A new medication made available last year offers these patients access to an oral formulation of ribavirin in combination with interferon alfa-2b. This new treatment option has higher sustained response rates than alfa interferon alone.
Another new compound, epivir-HBV tablets and oral solution, provides the first orally available treatment for chronic hepatitis B infection. Approval of this drug makes available an important therapeutic alternative to interferon, which is administered subcutaneously.
Children Children also benefited from 1998 approvals, which include seven products that can be used by young people.
Some of the most vulnerable victims of the AIDS virus -- little children -- now can get urgently needed help from three medications approved by CDER, one of which is for pediatric and two for both adult and pediatric use.
Nevirapine provides the first pediatric (liquid) formulation among this class of anti-HIV drugs. Efavirenz, one of the two therapies for both adults and children, has been shown to be effective in combination with other agents in suppressing the HIV virus for at least two years in patients as young as 3 years of age. Abacavir, the third new antiretroviral product, is an oral medication that also helps lower the amount of HIV in the blood, and can be taken by children as young as three months of age.
In addition, physicians now have four new products, two of which are vaccines, that have been found safe and effective in children.
One of the vaccines combats rotavirus, the leading cause of severe childhood diarrhea and dehydration that each year results in the hospitalization of 55,000 children. The other one, a genetically engineered protein, helps prevent Lyme disease and has been approved for use from the age 15 years and up.
The third product is palivizumab, a monoclonal antibody designed to protect high-risk infants against the respiratory syncytial virus (RSV) disease, the most common cause of lower respiratory infections in children. The disease each year puts more than 90,000 children into hospitals, about 4,500 of whom die.
The fourth drug is midazolam syrup. The product offers an advantage over the previous intravenous-only compound for children who need sedation, anxiolysis and amnesia prior to diagnostic, therapeutic or endoscopic procedures, or before induction of anesthesia. The approval of this product should also reduce the need for extemporaneous compounding of a liquid formulation of this drug.
Finally, CBER approved pediatric indications for two commercially available products: dornase alfa for use in treating cystic fibrosis in children 3 months to 4 years of age, and interferon alfa-2b for treatment of hepatitis B in pediatric populations.
Older Americans CDER's approvals last year included a drug designed for the 10-15 million American men, many of them elderly, who are affected by erectile dysfunction. Sildenafil is an oral therapy for a condition that previously could only be treated locally with injections, pellets or penile implants. The drug includes augmented labeling to emphasize its risks for patients with a history of coronary problems and abnormal blood pressure, and a contraindication for use with medications containing nitrates.
Other new treatments for the elderly include tolterodine, which offers the first new pharmaceutical therapy for incontinence in several decades, and brinzolamide, another treatment regimen for elevated intraocular pressure and open-angle glaucoma.
The diagnosis of osteoporosis, a significant weakening of bone that affects mostly the more than 20 million post-menopausal women in the U.S., was advanced by a new invention evaluated by CDRH. The device, the first such diagnostic instrument that does not use x-rays, estimates bone strength by transmitting high frequency sound waves through the patient's heel for about 10 seconds, and automatically analyzes the results. Early assessment of the risk of bone fracture is important for effective preventive measures and treatment.
Other Important Approvals In addition to efavirenz and acabavir, people with AIDS and cytomegalovirus infection (CMV) of the retina now have available a new biotechnology product that uses the first anti-sense approach to disease treatment approved by FDA. CMV is a major cause of blindness in patients with AIDS.
Thousands of Americans -- mostly men -- with coronary artery disease suffer disabling angina (chest pains) that cannot be adequately controlled by medications or effectively treated by balloon angioplasty or other surgical methods. A laser approved by CDRH last year can provide these patients with an otherwise unobtainable improvement in their condition by making tiny holes in the heart.
After many years during which pulmonary tuberculosis was a rarity in the U.S., 19,000 cases were reported in 1997. Last year, CDER approved rifapentine, the first new medicine for this indication in a quarter of a century. The new medication can be taken in lower doses and in less frequent intervals than older products, thereby encouraging patient compliance.
CBER's contribution to the fight against tuberculosis is a new indication for BCG vaccine for immunization against tuberculosis in adults and children over 12 months of age and in infants under 12 months.
Another newly approved product that serves a few, but very ill, patients is thalidomide. Two generations ago, FDA banned it from the U.S. market, thereby preventing the tragic fetal deformities that beset thousands of victims elsewhere. Last July, CDER used unprecedented safeguards to clear the drug as treatment for Hansen's disease (leprosy), which affects about 100 people in the U.S. The approval, based on a wealth of data showing that the drug improves skin lesions in at least 70-80 percent of patients, restricts the prescribing of thalidomide to selected physicians, and its use only to patients who agree to comply with stringent protective measures, including a patient registry.
FDA's Performance The products discussed in this report represent only a small part of the performance of CDER, CBER and CDRH. Both CDER and CBER exceeded the review performance goals of the prescription drugs user fee program that was reauthorized for another five years by the FDA Modernization Act of 1997. All of these reviews, however, did not result in approvals. Approximately one-half of the applications had to be returned to the manufacturers for further data and questions on deficiencies.
Like all interventions, the new products offer substantial benefits to patients, but also have certain risks. In many cases, the resulting approvals are highly restricted and subject to special warnings to inform the patient about potentially serious side effects. FDA remains vigilant to the appearance of new risks, and will continue to assess data to make sure that each product's benefits generally outweigh the known risks when used as directed.
CDER took 199 actions on original new drug applications (NDAs), 25 of which were for priority products considered of potentially exceptional public health value. Ninety of these actions were approvals completed in the median approval time of 12.0 months; the median time for approvals of the 25 priority products was 6.4 months. The cohort included 30 new chemical entities which were approved in the median total time of 12.0 months.
CBER took 80 actions on license application for both user fee and non-user fee products, 14 of which were for priority products. Thirty-two of the actions were approvals (completed in the median time of 13.4 months), and they included nine approvals for priority products whose median time to completion was 6.9 months. One license application was not approved, and six were withdrawn by the manufacturer.
CDRH, which does not receive any user fees, approved in fiscal year 1998 (10/1/97-9/30/98) 46 premarket approval applications (PMAs) for novel devices, 11 of which represented important diagnostic or therapeutic advances. CDRH maintained for the second year a backlog-free record of reviewing PMAs, PMA supplements, and 510(k)s, submissions for products similar to devices already on the market. The average time from submission to approval for PMAs in FY 98 was 12.4 months.