Practice Guidelines

Significant FDA Approvals in 2001

Am Fam Physician. 2002 Jun 1;65(11):2377-2383.

The most urgent task faced by the U.S. Food and Drug Administration (FDA) in the wake of the terrorist attacks on September 11, 2001, was to assure the availability of medical products for the treatment of injuries that could be caused by terrorists using biologic, chemical, or nuclear agents. Responding to this challenge, the agency's medical product centers—the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH)—rapidly launched numerous initiatives.

CDER clarified that the antibiotics doxycycline and penicillin G procaine are effective and approved for use in treating all forms of anthrax. CBER worked closely with industry and other government agencies to assure an adequate supply of products for immunization against anthrax, botulinum, small pox, and similar substances. CDRH contributed to the development of methodology for the detection of biologic agents that could be used for bioterrorism.

Product Approvals

The counter-terrorism programs were carried out in addition to the FDA's routine review and approval of medical products, which in 2001 included a breakthrough drug for cancer, the first biologic treatment for the most serious type of sepsis, and an advanced device for monitoring glucose levels in blood. CDER approved 66 new drugs, 24 of which were new molecular entities with ingredients not previously marketed in the United States (Table 1). CBER approved 16 complex biologic license applications, most of which were designed to detect or treat infections (see Table 2). CDRH approved 54 premarket approvals, of which 24 were for devices with novel technologies or new uses.

TABLE 1

Significant New Molecular Entity Drugs Approved in 2001

Drug Indications

Caspofungin acetate (Cancidas)

Injection for the treatment of invasive aspergillosis in patients who are refractory to or intolerant of other therapies

Ziprasidone hydrochloride (Geodon)

For the treatment of schizophrenia

Formoterol fumarate (Foradil Aerolizer)

Capsules or inhalation powder for maintenance treatment of asthma and for prevention of bronchospasm in adults and children five years and older; also used for acute prevention of exercise-induced bronchospasm in adults and children 12 years and older

Galantamine hydrobromide (Reminyl)

For the treatment of mild to moderate dementia of the Alzheimer's type

Travoprost (Travatan)

Ophthalmic solution for the reduction of intraocular pressure in patients with open-angle glaucoma or ocular hypertension who are intolerant of or insufficiently responsive to other intraocular pressure-lowering medications

Bimatoprost (Lumigan)

Ophthalmic solution for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension who are intolerant of or insufficiently responsive to other intraocular pressure-lowering medications

Almotriptan malate (Axert)

For the acute treatment of migraine

Imatinib mesylate (Gleevec)

For the treatment of patients with chronic myeloid leukemia in blast crisis, accelerated phase, or chronic phase after failure of interferon-alfa therapy

Drospirenone/ethinyl estradiol (Yasmin)

For oral contraception

Perflutren lipid microsphere (Definity)

Injectable agent for patients with suboptimal echocardiograms to opacify the left ventricular chamber and to improve the delineation of the left ventricular endocardial border

Nesiritide (Natrecor)

Intravenous treatment for patients with acutely decompensated congestive heart failure who have dyspnea at rest or with minimal activity

Zoledronic acid (Zometa)

Injectable treatment for hypercalcemia of malignancy

Cefditroen pivoxil (Spectracef)

For the treatment of acute bacterial exacerbation of chronic bronchitis, pharyngitis/tonsillitis, and uncomplicated skin and skin structure infections

Etonogestrel/ethinyl estradiol (NuvaRing)

Vaginal ring for the prevention of pregnancy

Tenofovir disoproxil fumarate (Viread)

Used in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults

Frovatriptan succinate (Frova)

For the acute treatment of migraine

Valdecoxib (Bextra)

For the relief of signs and symptoms of osteoarthritis and adult rheumatoid arthritis and for the treatment of primary dysmenorrhea

Norelgestromin/ethinyl estradiol (Ortho Evra)

Contraceptive patch for the prevention of pregnancy

Bosentan (Tracleer)

For the treatment of pulmonary arterial hypertension

Dutasteride (trade name is pending)

Soft-gelatin capsule for the treatment of symptomatic benign prostatic hyperplasia in men with an enlarged prostate gland

Ertapenem sodium (Invanz)

Injection indicated for the following: (1) complicated intra-abdominal infections; (2) complicated skin and skin structure infections; (3) community-acquired pneumonia; (4) complicated urinary tract infections, including pyelonephritis; and (5) acute pelvic infections, including postpartum endomyometritis, septic abortion, and postsurgical gynecologic infections

Fondaparinux sodium (Arixtra)

Injection indicated for the prophylaxis of deep venous thrombosis, which may lead to pulmonary embolism in patients undergoing (1) hip fracture surgery, (2) hip replacement surgery, and (3) knee replacement surgery

Pimecrolimus (Elidel)

Cream used for treatment of mild to moderate atopic dermatitis in non-immunocompromised patients two years and older who have contraindications or insufficient response to alternative, conventional therapies

Desloratadine (Clarinex)

For the relief of the nasal and non-nasal symptoms of seasonal allergic rhinitis in patients 12 years and older

TABLE 1   Significant New Molecular Entity Drugs Approved in 2001

View Table

TABLE 1

Significant New Molecular Entity Drugs Approved in 2001

Drug Indications

Caspofungin acetate (Cancidas)

Injection for the treatment of invasive aspergillosis in patients who are refractory to or intolerant of other therapies

Ziprasidone hydrochloride (Geodon)

For the treatment of schizophrenia

Formoterol fumarate (Foradil Aerolizer)

Capsules or inhalation powder for maintenance treatment of asthma and for prevention of bronchospasm in adults and children five years and older; also used for acute prevention of exercise-induced bronchospasm in adults and children 12 years and older

Galantamine hydrobromide (Reminyl)

For the treatment of mild to moderate dementia of the Alzheimer's type

Travoprost (Travatan)

Ophthalmic solution for the reduction of intraocular pressure in patients with open-angle glaucoma or ocular hypertension who are intolerant of or insufficiently responsive to other intraocular pressure-lowering medications

Bimatoprost (Lumigan)

Ophthalmic solution for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension who are intolerant of or insufficiently responsive to other intraocular pressure-lowering medications

Almotriptan malate (Axert)

For the acute treatment of migraine

Imatinib mesylate (Gleevec)

For the treatment of patients with chronic myeloid leukemia in blast crisis, accelerated phase, or chronic phase after failure of interferon-alfa therapy

Drospirenone/ethinyl estradiol (Yasmin)

For oral contraception

Perflutren lipid microsphere (Definity)

Injectable agent for patients with suboptimal echocardiograms to opacify the left ventricular chamber and to improve the delineation of the left ventricular endocardial border

Nesiritide (Natrecor)

Intravenous treatment for patients with acutely decompensated congestive heart failure who have dyspnea at rest or with minimal activity

Zoledronic acid (Zometa)

Injectable treatment for hypercalcemia of malignancy

Cefditroen pivoxil (Spectracef)

For the treatment of acute bacterial exacerbation of chronic bronchitis, pharyngitis/tonsillitis, and uncomplicated skin and skin structure infections

Etonogestrel/ethinyl estradiol (NuvaRing)

Vaginal ring for the prevention of pregnancy

Tenofovir disoproxil fumarate (Viread)

Used in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults

Frovatriptan succinate (Frova)

For the acute treatment of migraine

Valdecoxib (Bextra)

For the relief of signs and symptoms of osteoarthritis and adult rheumatoid arthritis and for the treatment of primary dysmenorrhea

Norelgestromin/ethinyl estradiol (Ortho Evra)

Contraceptive patch for the prevention of pregnancy

Bosentan (Tracleer)

For the treatment of pulmonary arterial hypertension

Dutasteride (trade name is pending)

Soft-gelatin capsule for the treatment of symptomatic benign prostatic hyperplasia in men with an enlarged prostate gland

Ertapenem sodium (Invanz)

Injection indicated for the following: (1) complicated intra-abdominal infections; (2) complicated skin and skin structure infections; (3) community-acquired pneumonia; (4) complicated urinary tract infections, including pyelonephritis; and (5) acute pelvic infections, including postpartum endomyometritis, septic abortion, and postsurgical gynecologic infections

Fondaparinux sodium (Arixtra)

Injection indicated for the prophylaxis of deep venous thrombosis, which may lead to pulmonary embolism in patients undergoing (1) hip fracture surgery, (2) hip replacement surgery, and (3) knee replacement surgery

Pimecrolimus (Elidel)

Cream used for treatment of mild to moderate atopic dermatitis in non-immunocompromised patients two years and older who have contraindications or insufficient response to alternative, conventional therapies

Desloratadine (Clarinex)

For the relief of the nasal and non-nasal symptoms of seasonal allergic rhinitis in patients 12 years and older

TABLE 2

Significant Biologic Approvals in 2001

Name Indications

Peginterferon alfa-2b (PEG-Intron)

Treatment of chronic hepatitis C in patients not previously treated with interferon alfa who have compensated liver disease and are at least 18 years of age

Alemtuzumab (Campath)

Treatment of patients with B-cell chronic lymphocytic leukemia who have been treated with alkylating agents and failed fludarabine therapy

Hepatitis A inactivated and hepatitis B (recombinant) vaccine (Twinrix)

Active immunization of persons 18 years or older against hepatitis A virus infection and all known subtypes of hepatitis B virus infection

Digoxin immune fab, ovine (DigiFab)

Treatment of patients with life-threatening or potentially life-threatening cardiac glycoside toxicity or overdose

Darbepoetin alfa (Aranesp)

Treatment of anemia associated with chronic renal failure, whether or not patient is on dialysis

UltraQual hepatitis C virus reverse transcriptase polymerase chain reaction (PCR) assay

A nucleic acid test used to detect HCV-RNA in pooled human source plasma

UltraQual human immunodeficiency virus type 1 reverse transcriptase PCR assay

A nucleic acid test used to detect HIV-1-RNA in pooled human source plasma

Hepatitis B immune globulin, human (Nabi-HB)

For postexposure prophylaxis of hepatitis infection

Anakinra (Kineret)

For reduction in signs and symptoms of moderately to severely active rheumatoid arthritis, in patients 18 years or older who have failed one or more disease modifying antirheumatic drugs

Drotrecogin alfa, activated (Xigris)

Reduction of mortality in adult patients with severe sepsis (sepsis associated with acute organ dysfunction) who have a high risk of death (e.g., as determined by APACHE II)

Acellular pertussis vaccine, concentrate

New formulation: preservative-free

Diphtheria and tetanus toxoids and acellular pertussis vaccine, adsorbed (Tripedia)

New formulation: preservative-free, single-dose vial (reduced thimerosal)

Basiliximab (Simulect)

Expanded indication: use in renal transplantation in combination with triple immunosuppressive therapy; use in pediatric renal transplantation; and use an intravenous bolus injection

Rituximab (Rituxan)

New dosing regimen: retreatment of patients with non-Hodgkin's lymphoma and administration of eight weekly doses; other changes to package insert: updated response data, data on treatment of bulky disease, warning about severe mucocutaneous reactions

Peginterferon alfa-2b (PEG-Intron)

Expanded indication: combination therapy with ribavirin, USP (Rebetol), for treatment of chronic hepatitis C

Alteplase (Cathflo Activase)

New indication and dosing strength: 2-mg vial strength for the restoration of function to central venous access devices (as assessed by the ability to withdraw blood)

TABLE 2   Significant Biologic Approvals in 2001

View Table

TABLE 2

Significant Biologic Approvals in 2001

Name Indications

Peginterferon alfa-2b (PEG-Intron)

Treatment of chronic hepatitis C in patients not previously treated with interferon alfa who have compensated liver disease and are at least 18 years of age

Alemtuzumab (Campath)

Treatment of patients with B-cell chronic lymphocytic leukemia who have been treated with alkylating agents and failed fludarabine therapy

Hepatitis A inactivated and hepatitis B (recombinant) vaccine (Twinrix)

Active immunization of persons 18 years or older against hepatitis A virus infection and all known subtypes of hepatitis B virus infection

Digoxin immune fab, ovine (DigiFab)

Treatment of patients with life-threatening or potentially life-threatening cardiac glycoside toxicity or overdose

Darbepoetin alfa (Aranesp)

Treatment of anemia associated with chronic renal failure, whether or not patient is on dialysis

UltraQual hepatitis C virus reverse transcriptase polymerase chain reaction (PCR) assay

A nucleic acid test used to detect HCV-RNA in pooled human source plasma

UltraQual human immunodeficiency virus type 1 reverse transcriptase PCR assay

A nucleic acid test used to detect HIV-1-RNA in pooled human source plasma

Hepatitis B immune globulin, human (Nabi-HB)

For postexposure prophylaxis of hepatitis infection

Anakinra (Kineret)

For reduction in signs and symptoms of moderately to severely active rheumatoid arthritis, in patients 18 years or older who have failed one or more disease modifying antirheumatic drugs

Drotrecogin alfa, activated (Xigris)

Reduction of mortality in adult patients with severe sepsis (sepsis associated with acute organ dysfunction) who have a high risk of death (e.g., as determined by APACHE II)

Acellular pertussis vaccine, concentrate

New formulation: preservative-free

Diphtheria and tetanus toxoids and acellular pertussis vaccine, adsorbed (Tripedia)

New formulation: preservative-free, single-dose vial (reduced thimerosal)

Basiliximab (Simulect)

Expanded indication: use in renal transplantation in combination with triple immunosuppressive therapy; use in pediatric renal transplantation; and use an intravenous bolus injection

Rituximab (Rituxan)

New dosing regimen: retreatment of patients with non-Hodgkin's lymphoma and administration of eight weekly doses; other changes to package insert: updated response data, data on treatment of bulky disease, warning about severe mucocutaneous reactions

Peginterferon alfa-2b (PEG-Intron)

Expanded indication: combination therapy with ribavirin, USP (Rebetol), for treatment of chronic hepatitis C

Alteplase (Cathflo Activase)

New indication and dosing strength: 2-mg vial strength for the restoration of function to central venous access devices (as assessed by the ability to withdraw blood)

Cancer Patients

One of the FDA's most important approvals last year was for imatinib mesylate or STI-571 (Gleevec), a new oral treatment for patients with chronic myeloid leukemia. In addition to two cancer drugs for women, the FDA approved alemtuzumab Campath, a new biologic product for the treatment of patients with B-cell chronic lymphocytic leukemia who have been treated with alkylating agents and failed fludarabine therapy.

Women and Children

The first of two cancer products for women, a combination of capecitabine (Xeloda) and docetaxel (Taxotere), was approved for the treatment of metastatic breast cancer that has not responded to an anthracycline-containing cancer therapy.

The other approval was a new indication for letrozole (Femara) as a first-line treatment for advanced or meta-static breast cancer in postmenopausal women with hormone receptor-positive or unknown disease.

In addition, two contraceptive devices that deliver continuous doses of progestin and estrogen hormones were approved. One of the devices, Ortho Evra, is the first skin patch approved for birth control, and the other, Nuva-Ring, delivers the hormones through a flexible, transparent polymer ring that is inserted into the vagina.

The FDA also approved the first automatic external defibrillator (AED) system for use on infants and young children who experience cardiac arrest.

Heart Patients

In addition to the AED system for use on infants and young children, the FDA approved the following four highly advanced medical devices for heart patients: Biotronik Home Monitoring System, the first implanted pacemaker that includes a tiny transmitter capable of automatic, remote data transmission; the Wearable Cardioverter Defibrillator System, a vest-like device that is worn under clothing to monitor and treat abnormal heart rhythms in people at risk of dying from sudden cardiac arrest; InSync Biventricular Cardiac Pacing System, a new type of pacemaker that sends specially timed electric impulses to the heart's lower chambers to treat the symptoms of moderate to severe congestive heart failure; and PercuSurge, a device consisting of balloon and aspiration catheters, which is used to collect and remove blood clots and other debris created by angioplasty and stenting of a blocked bypass vein graft.

Nesiritide (Natrecor), a new drug approved for cardiac patients, is an injection treatment for acute congestive heart failure. The medication, which was developed with the use of recombinant DNA technology, is a synthetic version of human hormone that dilates veins and arteries.

Infectious Diseases

Xigris is the first biologic treatment approved for the most serious forms of life-threatening sepsis, which claims 225,000 lives in the United States each year. The new treatment is a genetically engineered version of a naturally occurring human protein, Activated Protein C, which interferes with some of the body's harmful responses to severe infection.

Peginteferon alfa-2b (PEG-Intron) injection was approved for the treatment of patients with chronic hepatitis C, an infectious disease responsible for as many as 10,000 deaths per year in the United States. PEG-Intron is a longer acting form of interferon than Intron A (a previously approved form of interferon) and requires only one injection per week for one year compared with three per week for Intron A. The FDA also approved a supplemental application for the use of PEG-Intron with ribavirin (Rebetol) capsules, which was shown to be somewhat more effective than Intron A with Rebetol.

In addition, the FDA approved Twinrix, a new combination vaccine that protects adults against diseases caused by the hepatitis A virus (HAV) and the hepatitis B virus (HBV). Twinrix is recommended for travelers who are at high risk for HBV and are visiting countries where there is a substantial incidence of HAV and HBV disease.

Caspofungin acetate (Cancidas) intravenous infusion is a new antifungal medication for patients not responsive to or unable to tolerate standard therapies for the invasive form of aspergillosis. This is the first approval in a new class of drugs called echinocandins, which are believed to work by disrupting the creation of fungal cell walls.

Tenofovir disoproxil fumarate (Viread) is a new antiviral drug for the treatment of human immunodeficiency virus-1 (HIV-1) infection in combination with other anti-retroviral medicines. Viread is the first nucleotide analog approved for HIV-1 treatment.

Other Important Approvals

Bimatoprost ophthalmic solution (Lumigan) and travoprost ophthalmic solution (Travatan) are new medications to treat the elevated intraocular pressure often associated with glaucoma. The solutions are indicated for use in patients who are intolerant of other intraocular pressure-lowering medications.

Darbepoetin (Aranesp), a new biologic agent, was approved for treatment of anemia associated with chronic renal failure, whether or not patients are on dialysis.

The agency also approved the first HIV drug resistance test for use as an aid in monitoring and treating HIV-infected patients. The test detects mutations in HIV that confer resistance to drugs used to treat the infection.

Novel devices approved in 2001 included GlucoWatch Biographer, a wristwatch-like device that provides adult diabetics with more information for managing their disease, and Lap-Band Adjustable Gastric Banding System, an inflatable device that is surgically placed around the upper stomach to limit food consumption and create an earlier feeling of fullness. This device is intended for use in people at least 100 lb overweight who have failed to reduce their weight by dieting and other methods, and who are at greater risk for serious diseases such as hypertension, gall bladder disease, and diabetes. The FDA also cleared a new device that produces images that help detect bleeding of the small bowel inner lining.

The complete new drug approval tables are available at this Web site: www.fda.gov.

Thomas J. McGinnis, R.Ph., is director of pharmacy affairs in the Office of Policy of the U.S. Food and Drug Administration.


Copyright © 2002 by the American Academy of Family Physicians.
This content is owned by the AAFP. A person viewing it online may make one printout of the material and may use that printout only for his or her personal, non-commercial reference. This material may not otherwise be downloaded, copied, printed, stored, transmitted or reproduced in any medium, whether now known or later invented, except as authorized in writing by the AAFP. Contact afpserv@aafp.org for copyright questions and/or permission requests.

Want to use this article elsewhere? Get Permissions


Article Tools

  • Print page
  • Share this page
  • AFP CME Quiz

Information From Industry

More in Pubmed

Navigate this Article