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U.S. Food and Drug Administration • Center for Drug Evaluation and Research |
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Orphan Drug Designation The Orphan Drug Act provides for granting special status, called Orphan Designation, to the combination of a product and indication if requested by a sponsor and if certain criteria are met. Orphan designation qualifies the sponsor of the product for tax credits and marketing exclusivity incentives in addition to direct financial aid to assist in the clinical development. Further details are available through the Office of Orphan Products Development at the FDA. Listed below are oncology drugs and the approved use that in combination have received Orphan Designation. |
| Generic Name | Trade Name | Approved Use | Manufacturer/ Distributor | Approval Date |
|---|---|---|---|---|
| Aldesleukin | Proleukin | Treatment of adults with metastatic renal cell carcinoma | Chiron Corp | 05/05/1992 |
| Alemtuzumab | Campath | Accel. Approv. (clinical benefit not established) Campath is indicated for the treatment of B-cell chronic lymphocytic leukemia (B-CLL) in patients who have been treated with alkylating agents and who have failed fludarabine therapy. | Millennium and ILEX Partners, LP | 05/07/2001 |
| alitretinoin | Panretin | Topical treatment of cutaneous lesions in patients with AIDS-related Kaposi's sarcoma. | Ligand Pharmaceuticals | 02/02/1999 |
| allopurinol | Zyloprim | Patients with leukemia, lymphoma and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy. | GlaxoSmithKline | 05/17/1996 |
| altretamine | Hexalen | Single agent palliative treatment of patients with persistent or recurrent ovarian cancer following first-line therapy with a cisplatin and/or alkylating agent based combination. | US Bioscience | 12/26/1990 |
| amifostine | Ethyol | To reduce the cumulative renal toxicity associated with repeated administration of cisplatin in patients with advanced ovarian cancer | US Bioscience | 12/08/1995 |
| amifostine | Ethyol | Accel. Approv. (clinical benefit not established) Reduction of platinum toxicity in non-small cell lung cancer | US Bioscience | 03/15/1996 |
| amifostine | Ethyol | To reduce post-radiation xerostomia for head and neck cancer where the radiation port includes a substantial portion of the parotid glands. | US Bioscience | 06/24/1999 |
| arsenic trioxide | Trisenox | Second line treatment of relapsed or refractory APL following ATRA plus an anthracycline. | Cell Therapeutic | 09/25/2000 |
| bexarotene capsules | Targretin | For the treatment by oral capsule of cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to at least one prior systemic therapy. | Ligand Pharmaceuticals | 12/29/1999 |
| bexarotene gel | Targretin | For the topical treatment of cutaneous manifestations of cutaneous T-cell lymphoma in patients who are refractory to at least one prior systemic therapy. | Ligand Pharmaceuticals | 06/28/2000 |
| bleomycin | Blenoxane | Bristol-Myers Squibb | 07/31/1973 | |
| bleomycin | Blenoxane | Sclerosing agent for the treatment of malignant pleural effusion (MPE) and prevention of recurrent pleural effusions. | Bristol-Myers Squibb | 02/20/1996 |
| busulfan intravenous | Busulfex | Use in combination with cyclophoshamide as conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia. | Orphan Medical, Inc | 02/04/1999 |
| carmustine | BCNU, BiCNU | Bristol-Myers Squibb | 03/07/1977 | |
| carmustine with Polifeprosan 20 Implant | Gliadel Wafer | For use in addition to surgery to prolong survival in patients with recurrent glioblastoma multiforme who qualify for surgery. | Guilford Pharmaceuticals Inc. | 09/23/1996 |
| cladribine | Leustatin, 2-CdA | Treatment of active hairy cell leukemia. | R.W. Johnson Pharmaceutical Research Institute | 02/26/1993 |
| cytarabine liposomal | DepoCyt | Accel. Approv. (clinical benefit not established) Intrathecal therapy of lymphomatous meningitis | Skye Pharmaceuticals | 04/01/1999 |
| Denileukin diftitox | Ontak | Accel. Approv. (clinical benefit not established) treatment of patients with persistent or recurrent cutaneous T-cell lymphoma whose malignant cells express the CD25 component of the IL-2 receptor | Seragen, Inc | 02/05/1999 |
| dexrazoxane | Zinecard | Conversion to regular approval for reducing the incidence and severity of cardiomyopathy associated with doxorubicin administration in women with metastatic breast cancer who have received a cumulative doxorubicin dose of 300 mg/m2 and who will continue to receive doxorubicin therapy to maintain tumor control. It is not recommended for use with the initiation of doxorubicin therapy. | Pharmacia & Upjohn Company | 10/31/2002 |
| dexrazoxane | Zinecard | Accel. Approv. (clinical benefit subsequently established) Prevention of cardiomyopathy associated with doxorubicin administration | Pharmacia & Upjohn Company | 05/26/1995 |
| docetaxel | Taxotere | For locally advanced or metastatic non-small cell lung cancer after failure of prior platinum-based chemotherapy. | Aventis Pharmaceutical | 12/23/1999 |
| doxorubicin liposomal | Doxil | Conversion to regular approval for treatment of patients with ovarian cancer whose disease has progressed or recurred after platinum-based chemotherapy | Alza | 01/28/2005 |
| doxorubicin liposomal | Doxil | Accel. Approv. (clinical benefit not established) Treatment of metastatic carcinoma of the ovary in patient with disease that is refractory to both paclitaxel and platinum based regimens | Sequus Pharmaceuticals, Inc. | 06/28/1999 |
| doxorubicin liposomal | Doxil | Accel. Approv. (clinical benefit not established) Treatment of AIDS-related Kaposi's sarcoma in patients with disease that has progressed on prior combination chemotherapy or in patients who are intolerant to such therapy. | Sequus Pharmaceuticals, Inc. | 11/17/1995 |
| Elliott's B Solution | Elliott's B Solution | Diluent for the intrathecal administration of methotrexate sodium and cytarabine for the prevention or treatment of meningeal leukemia or lymphocytic lymphoma. | Orphan Medical, Inc | 09/27/1996 |
| epirubicin | Ellence | A component of adjuvant therapy in patients with evidence of axillary node tumor involvement following resection of primary breast cancer. | Pharmacia & Upjohn Company | 09/15/1999 |
| Epoetin alfa | epogen | EPOGENB is indicated for the treatment of anemia in patients with non-myeloid malignancies where anemia is due to the effect of concomitantly administered chemotherapy. EPOGEND is indicated to decrease the need for transfusions in patients who will be receiving concomitant chemotherapy for a minimum of 2 months. EPOGENB is not indicated for the treatment of anemia in cancer patients due to other factors such as iron or folate deficiencies, hemolysis or gastrointestinal bleeding, which should be managed appropriately. | Amgen, Inc | 07/26/1999 |
| Epoetin alfa | epogen | EPOGENB is indicated for the reatment of anemia related to therapy with zidovudine in HIV- infected patients. EPOGENB is indicated to elevate or maintain the red blood cell level (as manifested by the hematocrit or hemoglobin determinations) and to decrease the need for transfusions in these patients. EPOGEND is not indicated for the treatment of anemia in HIV-infected patients due to other factors such as iron or folate deficiencies, hemolysis or gastrointestinal bleeding, which should be managed appropriately. | Amgen, Inc | 07/26/1999 |
| Epoetin alfa | epogen | EPOGENB is indicated for the treatment of anemic patients (hemoglobin > 10 to _< 13 g/dL) scheduled to undergo elective, noncardiac, nonvascular surgery to reduce the need for allogeneic blood transfusions. | Amgen, Inc | 07/26/1999 |
| Epoetin alfa | epogen | EPOGEN is indicated for the treatment of anemia associated with CRF, including patients on dialysis (ESRD) and patients not on dialysis. | Amgen, Inch | 07/26/1999 |
| exemestane | Aromasin | Treatment of advance breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy. | Pharmacia & Upjohn Company | 10/21/1999 |
| Filgrastim | Neupogen | Decrease incidence of infection in patients with nonmyeloid malignancies | Amgen, Inc | 02/20/1991 |
| Filgrastim | Neupogen | NEUPOGEN is indicated to decrease the incidence of infection‚ as manifested by febrile neutropenia‚ in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with a significant incidence of severe neutropenia with fever. | Amgen, Inc | 04/02/1998 |
| Filgrastim | Neupogen | NEUPOGEN is indicated for reducing the time to neutrophil recovery and the duration of fever, following induction or consolidation hemotherapy treatment of adults with AML. | Amgen, Inc | 04/02/1998 |
| Filgrastim | Neupogen | NEUPOGEN is indicated to reduce the duration of neutropenia and neutropenia-related clinical sequelae, eg, febrile neutropenia, in patients with nonmyeloid malignancies undergoing myeloablative chemotherapy followed by marrow transplantation. | Amgen, Inc | 04/02/1998 |
| fludarabine | Fludara | Palliative treatment of patients with B-cell lymphocytic leukemia (CLL) who have not responded or have progressed during treatment with at least one standard alkylating agent containing regimen. | Berlex Laboratories Inc. | 04/18/1991 |
| fluorouracil, 5-FU | Adrucil | prolong survival in combination with leucovorin | ICN Puerto Rico | 04/25/1962 |
| gemtuzumab ozogamicin | Mylotarg | Accel. Approv. (clinical benefit not established) Treatment of CD33 positive acute myeloid leukemia in patients in first relapse who are 60 years of age or older and who are not considered candidates for cytotoxic chemotherapy. | Wyeth Ayerst | 05/17/2000 |
| hydroxyurea | Hydrea | Bristol-Myers Squibb | 12/07/1967 | |
| hydroxyurea | Hydrea | Decrease need for transfusions in sickle cell anemia | Bristol-Myers Squibb | 02/25/1998 |
| idarubicin | Idamycin | For use in combination with other approved antileukemic drugs for the treatment of acute myeloid leukemia (AML) in adults. | Adria Laboratories | 09/27/1990 |
| idarubicin | Idamycin | In combination with other approved antileukemic drugs for the treatment of acute non-lymphocytic leukemia in adults. | Pharmacia & Upjohn Company | 02/17/1997 |
| ifosfamide | IFEX | Third line chemotherapy of germ cell testicular cancer when used in combination with certain other approved antineoplastic agents. | Bristol-Myers Squibb | 12/30/1988 |
| imatinib mesylate | Gleevec | Accel. Approv. (clinical benefit not established) Initial therapy of chronic myelogenous leukemia | Novartis | 05/10/2001 |
| imatinib mesylate | Gleevec | Accel. Approv. (clinical benefit not established) Initial treatment of newly diagnosed Ph+ chronic myelogenous leukemia (CML). | Novartis | 12/20/2002 |
| imatinib mesylate | Gleevec | Accel. Approv. (clinical benefit not established) metastatic or unresectable malignant gastrointestinal stromal tumors | Novartis | 02/01/2002 |
| Interferon alfa-2a | Roferon-A | Hoffmann-La Roche Inc | 11/01/1996 | |
| Interferon alfa-2b | Intron A | Interferon alfa-2b, recombinant for Injection is indicated for intralesional treatment of selected patients 18 years of age or older with condylomata acuminata involving external surfaces of the genital and perianal areas. | Schering Corp | 06/06/1988 |
| Interferon alfa-2b | Intron A | Interferon alfa-2b, recombinant for Injection is indicated for the treatment of patients 18 years of age or older with hairy cell leukemia. | Schering Corp | 06/04/1986 |
| Interferon alfa-2b | Intron A | Interferon alfa-2b, recombinant for injection is indicated as adjuvant to surgical treatment in patients 18 years of age or older with malignant melanoma who are free of disease but at high risk for systemic recurrence within 56 days of surgery. | Schering Corp | 12/05/1995 |
| Interferon alfa-2b | Intron A | Interferon alfa-2b, recombinant for Injection is indicated for the initial treatment of clinically aggressive follicular non-Hodgkin’s Lymphoma in conjunction with anthracycline-containing combination chemotherapy in patients 18 years of age or older. | Schering Corp | 11/06/1997 |
| Interferon alfa-2b | Intron A | Interferon alfa-2b, recombinant for injection is indicated for the treatment of selected patients 18 years of age or older with AIDS-related Kaposi's Sarcoma. The likelihood of response to INTRON A therapy is greater in patients who are without systemic symptoms, who have limited lymphadenopathy and who have a relatively intact immune system as indicated by total CD4 count. | Schering Corp | 11/21/1988 |
| leucovorin | Wellcovorin, Leucovorin | Leucovorin calcium is indicated fro use in combination with 5-fluorouracil to prolong survival in the palliative treatment of patients with advanced colorectal cancer. | Immunex Corporation | 06/20/1952 |
| leucovorin | Leucovorin | Immunex Corporation | 01/30/1987 | |
| leucovorin | Leucovorin | Immunex Corporation | 01/30/1987 | |
| leucovorin | Leucovorin | Immunex Corporation | 08/31/1988 | |
| leucovorin | Leucovorin | In combination with fluorouracil to prolong survival in the palliative treatment of patients with advanced colorectal cancer. | Lederle Laboratories | 12/12/1991 |
| megestrol acetate | Megace | Bristol-Myers Squibb | 08/18/1971 | |
| melphalan, L-PAM | Alkeran | Systemic administration for palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. | GlaxoSmithKline | 11/18/1992 |
| mesna | Mesnex | Prevention of ifosfamide-induced hemorrhagic cystitis | Asta Medica | 12/30/1988 |
| methotrexate | Methotrexate | Lederle Laboratories | 08/10/1959 | |
| methotrexate | Methotrexate | osteosarcoma | Lederle Laboratories | 04/07/1988 |
| methotrexate | Methotrexate | Lederle Laboratories | 12/07/1953 | |
| methotrexate | Methotrexate | Lederle Laboratories | 10/31/1988 | |
| methotrexate | Methotrexate | Lederle Laboratories | 11/01/1971 | |
| methotrexate | Methotrexate | Lederle Laboratories | 11/01/1971 | |
| methoxsalen | Uvadex | For the use of UVADEX with the UVAR Photopheresis System in the palliative treatment of the skin manifestations of cutaneous T-cell lymphoma (CTCL) that is unresponsive to other forms of treatment. | Therakos | 02/25/1999 |
| mitoxantrone | Novantrone | For use in combination with corticosteroids as initial chemotherapy for the treatment of patients with pain related to advanced hormone-refractory prostate cancer. | Immunex Corporation | 11/13/1996 |
| mitoxantrone | Novantrone | For use with other approved drugs in the initial therapy for acute nonlymphocytic leukemia (ANLL) in adults. | Lederle Laboratories | 12/23/1987 |
| Oprelvekin | Neumega | Prevention of severe thrombocytopenia following myelosuppressive chemotherapy | Genetics Institute, Inc | 11/25/1997 |
| Oprelvekin | Neumega | Genetics Institute, Inc | 09/18/2002 | |
| Oprelvekin | Neumega | Neumega is indicated for the prevention of severe thrombocytopenia and the reduction of the need for platelet transfusions following myelosuppressive chemotherapy in adult patients with nonmyeloid malignancies who are at high risk of severe thrombocytopenia. | Genetics Institute, Inc | 09/18/2002 |
| oxaliplatin | Eloxatin | Accel. Approv. (clinical benefit not established) in combination with infusional 5-FU/LV, is indicated for the treatment of patients with metastatic carcinoma of the colon or rectum whose disease has recurred or progressed during or within 6 months of completion of first line therapy with the combination of bolus 5-FU/LV and irinotecan. | Sanofi Synthelabo | 08/09/2002 |
| paclitaxel | Paxene | treatment of advanced AIDS-related Kaposi's sarcoma after failure of first line or subsequent systemic chemotherapy | Baker Norton Pharmaceuticals, Inc | 12/24/1997 |
| paclitaxel | Taxol | For the adjuvant treatment of node-positive breast cancer administered sequentially to standard doxorubicin-containing combination therapy. | Bristol-Myers Squibb | 10/25/1999 |
| paclitaxel | Taxol | Treatment of patients with metastatic carcinoma of the ovary after failure of first-line or subsequent chemotherapy. | Bristol-Myers Squibb | 12/29/1992 |
| paclitaxel | Taxol | First line ovarian cancer with 3 hour infusion. | Bristol-Myers Squibb | 06/20/2000 |
| paclitaxel | Taxol | New dosing regimen for patients who have failed initial or subsequent chemotherapy for metastatic carcinoma of the ovary | Bristol-Myers Squibb | 06/22/1994 |
| paclitaxel | Taxol | second line therapy for AIDS related Kaposi's sarcoma. | Bristol-Myers Squibb | 08/04/1997 |
| paclitaxel | Taxol | For first-line therapy for the treatment of advanced carcinoma of the ovary in combination with cisplatin. | Bristol-Myers Squibb | 04/09/1998 |
| paclitaxel | Taxol | for use in combination with cisplatin, for the first-line treatment of non-small cell lung cancer in patients who are not candidates for potentially curative surgery and/or radiation therapy. | Bristol-Myers Squibb | 06/30/1998 |
| paclitaxel | Taxol | Treatment of breast cancer after failure of combination chemotherapy for metastatic disease or relapse within 6 months of adjuvant chemotherapy. Prior therapy should have included an anthracycline unless clinically contraindicated. | Bristol-Myers Squibb | 04/13/1994 |
| pegaspargase | Oncaspar | Acute lymphocytic leukemia in L-asparaginase hypersensitive patients | Enzon, Inc | 02/01/1994 |
| pentostatin | Nipent | Single agent treatment for adult patients with alpha interferon refractory hairy cell leukemia. | Parke-Davis Pharmaceutical Co. | 10/11/1991 |
| pentostatin | Nipent | Single-agent treatment for untreated hairy cell leukemia patients with active disease as defined by clinically significant anemia, neutropenia, thrombocytopenia, or disease-related symptoms. (Supplement for front -line therapy.) | Parke-Davis Pharmaceutical Co. | 09/29/1993 |
| porfimer sodium | Photofrin | For use in photodynamic therapy for treatment of microinvasive endobronchial nonsmall cell lung cancer in patients for whom surgery and radiotherapy are not indicated. | QLT Phototherapeutics Inc. | 01/09/1998 |
| porfimer sodium | Photofrin | For use in photodynamic therapy (PDT) for reduction of obstruction and palliation of symptoms in patients with completely or partially obstructing endobroncial nonsmall cell lung cancer (NSCLC). | QLT Phototherapeutics Inc. | 12/22/1998 |
| porfimer sodium | Photofrin | For use in photodynamic therapy (PDT) for palliation of patients with completely obstructing esophageal cancer, or patients with partially obstructing esophageal cancer who cannot be satisfactorily treated with ND-YAG laser therapy. | QLT Phototherapeutics Inc. | 12/27/1995 |
| Rituximab | Rituxan | Treatment of patients with relapsed or refractory low-grade or follicular B-cell non-Hodgkin's lymphoma | Genentech, Inc | 11/26/1997 |
| Sargramostim | Prokine | Immunex Corp | 11/07/1996 | |
| talc | Sclerosol | For the prevention of the recurrence of malignant pleural effusion in symptomatic patients. | Bryan | 12/24/1997 |
| temozolomide | Temodar | Accel. Approv. (clinical benefit not established) Treatment of adult patients with refractory anaplastic astrocytoma, i.e., patients at first relapse with disease progression on a nitrosourea and procarbazine containing regimen | Schering | 08/11/1999 |
| teniposide, VM-26 | Vumon | In combination with other approved anticancer agents for induction therapy in patients with refractory childhood acute lymphoblastic leukemia (all). | Bristol-Myers Squibb | 07/14/1992 |
| toremifene | Fareston | Treatment of advanced breast cancer in postmenopausal women. | Orion Corp. | 05/29/1997 |
| Trastuzumab | Herceptin | Herceptin in combination with paclitaxel is indicated for treatment of patients with metastatic breast cancer whose tumors overexpress the HER-2 protein and had not received chemotherapy for their metastatic disease | Genentech, Inc | 02/09/2000 |
| Trastuzumab | Herceptin | Genentech, Inc | 08/28/2002 | |
| Trastuzumab | Herceptin | Genentech, Inc | 08/28/2002 | |
| Trastuzumab | Herceptin | HERCEPTIN as a single agent is indicated for the treatment of patients with metastatic breast cancer whose tumors overexpress the HER2 protein and who have received one or more chemotherapy regimens for their metastatic disease. | Genentech, Inc | 09/25/1998 |
| tretinoin, ATRA | Vesanoid | Induction of remission in patients with acute promyelocytic leukemia (APL) who are refractory to or unable to tolerate anthracycline based cytotoxic chemotherapeutic regimens. | Roche | 11/22/1995 |
| valrubicin | Valstar | For intravesical therapy of BCG-refractory carcinoma in situ (CIS) of the urinary bladder in patients for whom immediate cystectomy would be associated with unacceptable morbidity or mortality. | Anthra --> Medeva | 09/25/1998 |
| zoledronate | Zometa | the treatment of patients with multiple myeloma and patients with documented bone metastases from solid tumors, in conjunction with standard antineoplastic therapy. Prostate cancer should have progressed after treatment with at least one hormonal therapy | Novartis | 02/22/2002 |
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