ROCKVILLE, Md., Jan. 15 /PRNewswire/ -- The product review record of the
Food and Drug Administration in 1998 demonstrates the agency's exceptional
progress in making available safe and effective medications and devices that
reflect the finest aspirations of modern medicine. These products address
diseases that in the past had not yielded to the efforts of researchers; they
promise relief to a great variety of patient groups, including some that have
lacked meaningful options; and they provide new testimony to the benefits that
state-of-the-art science can bring to those who suffer.
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.
For details about the Centers' 1998 performance, see the following
reports.
Biologics Center's 1998 Performance
FDA's Center for Biologics Evaluation and Research in 1998 took 80
actions, one more than the year before, on license applications for both user
fee and non-user fee products. Fourteen (16 in 1997) of these applications
were for priority products. Thirty-two (35 in 1997) of the actions taken were
approvals, and they were completed in the median time of 13.4 months (11.1
months in 1997). Eight (7 in 1997) of the approvals were for priority products
with the median time to completion of 6.9 months (8.9 months in 1997). Six
(same number as in 1997) of the applications were withdrawn by the
manufacturers. The approved products included new technologies, production
methods and indications.
Among the user fee approvals, 11 were considered major and included both
product and biological license applications (PLAs and BLAs). In addition, CBER
approved nine major user fee PLA and BLA supplements. The PLA/BLA user fee
approvals were completed in the median time of 15.2 months (14.4 months in
1997). The median time for the approval of PLA/BLA supplements for user fee
products was 11.9 months (10.7 months in 1997).
Ten of the major approvals were first approvals of products containing a
substance or a combination of substances never before approved for the U.S.
market. The median time for first-time approvals in 1998 was 11.5 months (12
months in 1997).
In addition to the products included in FDA's Report on New Health Care
Products Approved in 1998, CBER approved two major products:
* Basiliximab which is used to prevent organ rejection in de novo renal
transplant recipient, and
* Antithromocyte globulin (Rabbit) for use to treat and prevent acute
organ rejection.
The complete list of CBER's major approvals for PLAs/BLAs and PLA/BLA
supplements follows:
PLAs/BLAs:
Human T-Lymphotropic virus types I & II
Fibrin sealant
Pooled plasma, solvent detergent treated
Basiliximab
Palivizumab
Diphteria & tetanus toxoids & acellular pertussis vaccine adsorbed
Infliximab
Rotavirus vaccine, live, oral, tetravalent
Trastuzumab
Etanercept
Lyme disease vaccine
Anti-thromocyte globulin (rabbit)
SOURCE Food and Drug Administration
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