FDA Perspective

Significant FDA Approvals in 2000

Am Fam Physician. 2001 May 15;63(10):2061-2064.

In 2000, the three medical product review staffs of the U.S. Food and Drug Administration (FDA)—the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health—approved 160 new drugs, biologic products and medical devices for use in the United States. Many of these approvals advance the prevention, diagnosis and treatment of serious and lifethreatening diseases. The most important drugs that passed the FDA review for safety and effectiveness are listed in Table 1. Biologic products are listed in Table 2.

Among the new products are the first drug providing treatment of vancomycin-resistant bacterial infections, a promising new surgical system that incorporates robotic technology and the first treatment that slows the progress of wet age-related macular degeneration. The following are some of the patient groups who will particularly benefit from these FDA approvals.

Cancer Patients

Several products approved in 2000 contribute to the prevention, early diagnosis or treatment of cancer. Three of these products are medical devices: the Optical Biopsy System, a laser system that improves the ability to distinguish small harmless growths from precancerous growths in the colon; the Senographe 2000D, the first mammography system that produces digital images on a solid-state receptor instead of analog images on a radiographic film; and the FocalSeal-L Surgical Sealant, a glue for sealing air leaks in lungs following the removal of cancerous tumors.

Among the new cancer medications, two were approved for cancers of the white blood cells. Arsenic trioxide (Trisenox) was found to be safe and effective in the treatment of acute promyelocytic leukemia, and gemtuzumab/ ozogamicin (Mylotarg) was approved for the treatment of CD33-positive acute myeloid leukemia in patients 60 years or older. In addition, tamoxifen citrate (Nolvadex) was approved for a new indication, to reduce the risk of invasive breast cancer in women with preinvasive cancer of the ducts.

Infants and Children

Several new products approved last year for pediatric and obstetric use were either specifically designed for the youngest patients or were adult drugs with pediatric indications.

The most advanced new products include the OxiFirst Fetal Oxygen Saturation Monitoring System, which measures oxygen saturation in the infant's blood as a sign of fetal health during labor and delivery; Prevnar, the first multivalent conjugate pneumococcal vaccine for infants and toddlers younger than age two; and budesonide inhalation suspension (Pulmicort Respules), the first antiinflammatory corticosteroid formulated for inhalation using a nebulizer in children between one and eight years of age. Another new pediatric drug is zanamivir (Relenza), for the treatment of uncomplicated influenza A and B in patients seven years and older.

Drugs approved in 2000 for adult and pediatric indications include oseltamivir (Tamiflu), an oral suspension for prophylaxis of influenza infection in adults and adolescents 13 years and older; a lopinavir/ritonavir combination (Kaletra), which received accelerated approval as a protease inhibitor for the treatment of human immunodeficiency virus; levothyroxine (Unithroid), the first single-ingredient oral levothyroxine product approved for the treatment of hypothyroidism; and tacrolimus (Protopic), an ointment for use in patients with moderate or severe eczema for whom standard eczema therapies present potential risks or who are not adequately treated by or do not tolerate standard eczema therapies.

TABLE 1

Significant New Molecular Entity Drugs Approved in 2000

Drug Indications

Cevimeline (Evoxac)

For the treatment of symptoms of dry mouth in patients with Sjögren's syndrome

Oxcarbazepine (Trileptal)

For use as monotherapy or adjunctive therapy in the treatment of partial seizures in adults with epilepsy and as adjunctive therapy in the treatment of partial seizures in children 4 to 16 years with epilepsy

Pantoprazole (Protonix)

For short-term treatment of erosive esophagitis associated with gastroesophageal reflux disease

Skin Exposure Reduction Paste Against Chemical Warfare Agents (SERPACWA)

Used only in conjunction with Mission Oriented Protective Posture (MOPP) gear to reduce or delay the absorption of chemical warfare agents through the skin when SERPACWA is applied before exposure

Zonisamide (Zonegran)

Used as an adjunctive therapy in the treatment of partial seizures in adults with epilepsy

Articaine, epinephrine (Septocaine)

For infiltration or nerve block anesthesia for dentistry

Verteporfin (Visudyne)

For the treatment of age-related macular degeneration in patients with predominantly classic subfoveal choroidal neovascularization

Meloxicam (Mobic)

For the relief of the signs and symptoms of osteoarthritis

Linezolid (Zyvox)

For the treatment of adults with vancomycin-resistant Enterococcus faecium infections, nosocomial pneumonia, complicated and uncomplicated skin and skin structure infections, and community-acquired pneumonia

Insulin glargine (Lantus)

For once-daily subcutaneous administration at bedtime in the treatment of adult and pediatric patients with type 1 diabetes mellitus or adult patients with type 2 diabetes mellitus who require basal (long-acting) insulin for the control of hyperglycemia

Rivastigmine (Exelon)

For the treatment of mild to moderate dementia associated with Alzheimer's disease

Gemtuzumab/ozogamicin (Mylotarg)

For the treatment of patients with CD33-positive acute myeloid leukemia in first relapse who are 60 years or older and who are not considered candidates for cytotoxic chemotherapy

Colesevelam (Welchol)

For the reduction of elevated low-density lipoprotein cholesterol, alone or in combination with an HMG-CoA reductase inhibitor, in patients with primary hypercholesterolemia (Frederickson type IIa)

Insulin aspart (NovoLog)

For the treatment of adults with diabetes mellitus; for the control of hyperglycemia

Triptorelin (Trelstar Depot)

For the palliative treatment of advanced prostate cancer

Argatroban (Acova)

Anticoagulant for prophylaxis or treatment of patients with thrombosis and heparin-induced thrombocytopenia

Tinzaparin (Innohep)

For the treatment of acute symptomatic deep venous thrombosis with or without pulmonary embolism when administered in conjunction with warfarin

Balsalazide (Colazal)

For the treatment of mild to moderate active ulcerative colitis

Docosanol (Abreva)

Topical cream for the treatment of cold sores and fever blisters

Unoprostone (Rescula)

For the lowering of intraocular pressure in patients with open-angle glaucoma or ocular hypertension who are intolerant of other intraocular pressure-lowering medications or insufficiently responsive (failed to achieve target pressure determined after multiple measurements over time) to another intraocular pressure-lowering medication

Cetrorelix (Cetrotide)

Injection for the prevention of premature luteinizing hormone surges in women undergoing controlled ovarian stimulation

Lopinavir/ritonavir (Kaletra)

Used in combination with other antiretroviral agents for the treatment of human immunodeficiency virus 1 infections in adults and pediatric patients six months and older

Arsenic trioxide (Trisenox)

For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression

Mifepristone (Mifeprex)

For medical termination of intrauterine pregnancy through 49 days of pregnancy

Bivalirudin (Angiomax)

Used as an anticoagulant in patients with unstable angina undergoing percutaneous transluminal coronary angioplasty

Nateglinide (Starlix)

Used as monotherapy and as an adjunct to diet and exercise to improve glycemic control in patients with type 2 diabetes, or used concomitantly with metformin to improve glycemic control

TABLE 1   Significant New Molecular Entity Drugs Approved in 2000

View Table

TABLE 1

Significant New Molecular Entity Drugs Approved in 2000

Drug Indications

Cevimeline (Evoxac)

For the treatment of symptoms of dry mouth in patients with Sjögren's syndrome

Oxcarbazepine (Trileptal)

For use as monotherapy or adjunctive therapy in the treatment of partial seizures in adults with epilepsy and as adjunctive therapy in the treatment of partial seizures in children 4 to 16 years with epilepsy

Pantoprazole (Protonix)

For short-term treatment of erosive esophagitis associated with gastroesophageal reflux disease

Skin Exposure Reduction Paste Against Chemical Warfare Agents (SERPACWA)

Used only in conjunction with Mission Oriented Protective Posture (MOPP) gear to reduce or delay the absorption of chemical warfare agents through the skin when SERPACWA is applied before exposure

Zonisamide (Zonegran)

Used as an adjunctive therapy in the treatment of partial seizures in adults with epilepsy

Articaine, epinephrine (Septocaine)

For infiltration or nerve block anesthesia for dentistry

Verteporfin (Visudyne)

For the treatment of age-related macular degeneration in patients with predominantly classic subfoveal choroidal neovascularization

Meloxicam (Mobic)

For the relief of the signs and symptoms of osteoarthritis

Linezolid (Zyvox)

For the treatment of adults with vancomycin-resistant Enterococcus faecium infections, nosocomial pneumonia, complicated and uncomplicated skin and skin structure infections, and community-acquired pneumonia

Insulin glargine (Lantus)

For once-daily subcutaneous administration at bedtime in the treatment of adult and pediatric patients with type 1 diabetes mellitus or adult patients with type 2 diabetes mellitus who require basal (long-acting) insulin for the control of hyperglycemia

Rivastigmine (Exelon)

For the treatment of mild to moderate dementia associated with Alzheimer's disease

Gemtuzumab/ozogamicin (Mylotarg)

For the treatment of patients with CD33-positive acute myeloid leukemia in first relapse who are 60 years or older and who are not considered candidates for cytotoxic chemotherapy

Colesevelam (Welchol)

For the reduction of elevated low-density lipoprotein cholesterol, alone or in combination with an HMG-CoA reductase inhibitor, in patients with primary hypercholesterolemia (Frederickson type IIa)

Insulin aspart (NovoLog)

For the treatment of adults with diabetes mellitus; for the control of hyperglycemia

Triptorelin (Trelstar Depot)

For the palliative treatment of advanced prostate cancer

Argatroban (Acova)

Anticoagulant for prophylaxis or treatment of patients with thrombosis and heparin-induced thrombocytopenia

Tinzaparin (Innohep)

For the treatment of acute symptomatic deep venous thrombosis with or without pulmonary embolism when administered in conjunction with warfarin

Balsalazide (Colazal)

For the treatment of mild to moderate active ulcerative colitis

Docosanol (Abreva)

Topical cream for the treatment of cold sores and fever blisters

Unoprostone (Rescula)

For the lowering of intraocular pressure in patients with open-angle glaucoma or ocular hypertension who are intolerant of other intraocular pressure-lowering medications or insufficiently responsive (failed to achieve target pressure determined after multiple measurements over time) to another intraocular pressure-lowering medication

Cetrorelix (Cetrotide)

Injection for the prevention of premature luteinizing hormone surges in women undergoing controlled ovarian stimulation

Lopinavir/ritonavir (Kaletra)

Used in combination with other antiretroviral agents for the treatment of human immunodeficiency virus 1 infections in adults and pediatric patients six months and older

Arsenic trioxide (Trisenox)

For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression

Mifepristone (Mifeprex)

For medical termination of intrauterine pregnancy through 49 days of pregnancy

Bivalirudin (Angiomax)

Used as an anticoagulant in patients with unstable angina undergoing percutaneous transluminal coronary angioplasty

Nateglinide (Starlix)

Used as monotherapy and as an adjunct to diet and exercise to improve glycemic control in patients with type 2 diabetes, or used concomitantly with metformin to improve glycemic control

TABLE 2

Significant Biologic Approvals in 2000

Name Indications

Pneumococcal 7-valent conjugate vaccine (Prevnar)

Immunization of infants two, four, six and 12 to 15 months of age to prevent invasive pneumococcal disease

Antihemophilic factor (ReFacto)

Control and prevention of hemorrhagic episodes and for short-term routine and surgical prophylaxis in patients with hemophilia A

BCG, live (PACIS)

Treatment of carcinoma-in-situ in the absence of associated invasive cancer of the bladder

Tenecteplase (TNKase)

Reduction of mortality associated with acute myocardial infarction

Anti-A (Murine Monoclonal); Anti-B (Murine Monoclonal); Anti-A and B (Murine Monoclonal), Anti A, B (MurineMonoclonal), Anti-D (Human Monoclonal) (IgM) (ID Micro Typing Systems)

First approval of monoclonal antibodies to detect ABO and D antigens in a gel test under a shared manufacturing agreement with Serologicals, LTD

Crotalidae Polyvalent Immune Fab (Ovine[CroFab])

Treatment of minimal and moderate North American Crotalidae envenomation

Botulinum Toxin Type B (MYOBLOC)

Treatment of cervical dystonia

Human Blood and Blood Components

First approval of a licensed applicant for variance from 21 CFR 640.3(d) and 21 CFR 640.3(f) for collection of blood and blood components from donors with hereditary hemochromatosis

Human Immunodeficiency Virus Type 1 and 2 (Synthetic Peptide) (Genetic Systems HIV-1/HIV-2 Peptide EIA)

Expanded indication: Testing of cadaveric serum samples

Interferon gamma-1b (Actimmune)

New indication: For delaying the time to disease progression in patients with severe, malignant osteoporosis

Autologous Cultured Chondrocytes (Carticel Service)

Revised indication: Narrowing the indication to second-line therapy, for use in patients who have had an inadequate response to a prior arthroscopic or other surgical repair procedure

Etanercept (Enbrel)

Expanded indication: Reducing the signs and symptoms and delaying structural damage in patients with moderately to severely active rheumatoid arthritis, including those who have not previously failed treatment with a disease-modifying antirheumatic drug

Antihemophilic Factor (Recombinant) (Kogenate FS)

New product formulation

Diptheria and Tetanus Toxoids and Acellular Pertussis Vaccine, Adsorbed (Tripedia)

Expanded patient population: Include a fifth dose at four to six years after four prior doses of Tripedia

Anti-D, Anti-A, Anti-B, Anti-A, B Monoclonal Antibodies

First approvals of monoclonal antibodies to detect ABO and D antigens in a gel test. For further manufacturing in a shared manufacturing agreement with MicroTyping Systems, Inc.

A/B/D Monoclonal Grouping Combination Gel Card (ID Micro Typing Systems)

First approval for production of a monoclonal grouping combination gel card

Botulinum Toxin Type A (BOTOX)

New indication: Treatment of cervical dystonia

Infliximab (Remicade)

Expanded indication: To include the inhibition of progression of structural damage in patients with rheumatoid arthritis who have had an inadequate response to methotrexate

Home-Access HIV-1 Test System2

HIV testing service to be used with the Organon Teknika Vironostika HIV-1 Microelisa System using the rapid elution protocol, allows test result availability within 24 hours

Roche Amplicor HIV-1 Monitor Test

Expanded indication: In vitro test for the quantitation of HIV-1 RNA in human plasma, allows monitoring patient and disease prognosis of HIV disease


BCG = bacille Calmette-Guérin; HIV = human immunodeficiency virus.

note:New Drug Applications (None in Calendar Year2000); Supplemental New Drug Applications (None in Calendar Year 2000).

TABLE 2   Significant Biologic Approvals in 2000

View Table

TABLE 2

Significant Biologic Approvals in 2000

Name Indications

Pneumococcal 7-valent conjugate vaccine (Prevnar)

Immunization of infants two, four, six and 12 to 15 months of age to prevent invasive pneumococcal disease

Antihemophilic factor (ReFacto)

Control and prevention of hemorrhagic episodes and for short-term routine and surgical prophylaxis in patients with hemophilia A

BCG, live (PACIS)

Treatment of carcinoma-in-situ in the absence of associated invasive cancer of the bladder

Tenecteplase (TNKase)

Reduction of mortality associated with acute myocardial infarction

Anti-A (Murine Monoclonal); Anti-B (Murine Monoclonal); Anti-A and B (Murine Monoclonal), Anti A, B (MurineMonoclonal), Anti-D (Human Monoclonal) (IgM) (ID Micro Typing Systems)

First approval of monoclonal antibodies to detect ABO and D antigens in a gel test under a shared manufacturing agreement with Serologicals, LTD

Crotalidae Polyvalent Immune Fab (Ovine[CroFab])

Treatment of minimal and moderate North American Crotalidae envenomation

Botulinum Toxin Type B (MYOBLOC)

Treatment of cervical dystonia

Human Blood and Blood Components

First approval of a licensed applicant for variance from 21 CFR 640.3(d) and 21 CFR 640.3(f) for collection of blood and blood components from donors with hereditary hemochromatosis

Human Immunodeficiency Virus Type 1 and 2 (Synthetic Peptide) (Genetic Systems HIV-1/HIV-2 Peptide EIA)

Expanded indication: Testing of cadaveric serum samples

Interferon gamma-1b (Actimmune)

New indication: For delaying the time to disease progression in patients with severe, malignant osteoporosis

Autologous Cultured Chondrocytes (Carticel Service)

Revised indication: Narrowing the indication to second-line therapy, for use in patients who have had an inadequate response to a prior arthroscopic or other surgical repair procedure

Etanercept (Enbrel)

Expanded indication: Reducing the signs and symptoms and delaying structural damage in patients with moderately to severely active rheumatoid arthritis, including those who have not previously failed treatment with a disease-modifying antirheumatic drug

Antihemophilic Factor (Recombinant) (Kogenate FS)

New product formulation

Diptheria and Tetanus Toxoids and Acellular Pertussis Vaccine, Adsorbed (Tripedia)

Expanded patient population: Include a fifth dose at four to six years after four prior doses of Tripedia

Anti-D, Anti-A, Anti-B, Anti-A, B Monoclonal Antibodies

First approvals of monoclonal antibodies to detect ABO and D antigens in a gel test. For further manufacturing in a shared manufacturing agreement with MicroTyping Systems, Inc.

A/B/D Monoclonal Grouping Combination Gel Card (ID Micro Typing Systems)

First approval for production of a monoclonal grouping combination gel card

Botulinum Toxin Type A (BOTOX)

New indication: Treatment of cervical dystonia

Infliximab (Remicade)

Expanded indication: To include the inhibition of progression of structural damage in patients with rheumatoid arthritis who have had an inadequate response to methotrexate

Home-Access HIV-1 Test System2

HIV testing service to be used with the Organon Teknika Vironostika HIV-1 Microelisa System using the rapid elution protocol, allows test result availability within 24 hours

Roche Amplicor HIV-1 Monitor Test

Expanded indication: In vitro test for the quantitation of HIV-1 RNA in human plasma, allows monitoring patient and disease prognosis of HIV disease


BCG = bacille Calmette-Guérin; HIV = human immunodeficiency virus.

note:New Drug Applications (None in Calendar Year2000); Supplemental New Drug Applications (None in Calendar Year 2000).

The FDA also added pediatric use to the labeling of 13 adult drugs, including Motrin, Advil, Zantac and Nasalcrom, and approved lowering the age for implantation of the Nucleus 24 Cochlear Implant from 18 months to 12 months in infants who are profoundly hearing impaired.

Women's Health

The FDA approved several products to treat diseases that either exclusively or predominantly affect women. They include infliximab (Remicade), for the reduction in signs and symptoms of rheumatoid arthritis, and the cancer drug mitoxantrone hydrochloride (Novantrone) for the treatment of advanced or chronic multiple sclerosis. The agency also allowed continued marketing of two types of saline-filled breast implants that had been approved for breast augmentation and breast reconstruction in women 18 years or older.

Cardiac Patients and the Elderly

New products for patients with heart disease and the elderly include several novel devices and important medications. The Cordis Checkmate System and the Novoste Beta-Cath System use catheters to deliver radiation inside a coronary stent to help reduce the risk of new tissue growth; tenecteplase (TNKase) is a new tissue plasminogen activator for the reduction of mortality in patients with acute myocardial infarction; and verteporfin for injection (Visudyne), the first therapy to slow vision loss caused by wet age-related macular degeneration. In 2000, the FDA also approved the Vibrant Soundbridge, a surgically implanted hearing device.

Other Significant Approvals

Linezolid (Zyvox) is the first antibacterial drug approved to treat infections associated with Enterococcus faecium resistant to vancomycin; ciprofloxacin (Cipro) was approved to reduce the incidence or progression of inhalational anthrax infection; the Da Vinci Surgical System is a first-ever robotic device for use in laparoscopic gall bladder surgery and surgery for reflux disease; and Trufill n-BCA Liquid Embolic System is a new glue approved to help reduce blood loss by blocking blood vessels in the brain during a rare form of brain surgery.

The FDA also approved Apligraf, a new type of wound dressing to help heal diabetic foot ulcers; botulinum toxin type B (Myobloc), the first drug to reduce the severity of neck and shoulder muscle contractions; Malarone, a new combination drug for the prevention and treatment of acute, uncom plicated Plasmodium falciparum malaria; Positron Emission Tomography (PET) F-18 FDG, a radiopharmaceutical drug that assists in cancer assessments and in identifying reversible cardiac dysfunction; and OssaTron, a device that uses shock waves to relieve chronic severe heel pain when conventional treatment has been ineffective.

Thomas J. McGinnis, R.Ph., is Director of Pharmacy Affairs in the Office of Policy for the U.S. Food and Drug Administration.

Coordinator of this quarterly series from the U.S. Food and Drug Administration is Mike Kubic of the Office of Public Affairs.


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