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FDA GRANTS ORPHAN STATUS TO FOUR AIDS TREATMENT DRUGS; WELLFERON IS THIRD BURROUGHS WELLCOME PRODUCT WITH ORPHAN INDICATION FOR AIDS ILLNESS

Executive Summary

Four drugs for treatment of AIDS-related illnesses are being developed under orphan drug designations made by FDA since last January, according to information released by FDA. The drugs are: Burroughs Wellcome's Wellferon (alfa-1 interferon) for treatment of Kaposi's sarcoma; Merrell Dow's eflornithine HCl (DFMO) and Warner-Lambert's trimetrexate gluconate for treatment of pneumocystis carinii pneumonia; and Merieux Institute's Imuthiol (diethyldithiocarbamate) for treatment of AIDS. Wellferon is among a group of Burroughs Wellcome compounds under investigation for AIDS related illnesses. The firm's AZT became the standard test drug for AIDS patients with pneumocystis carinii after preliminary results of a Phase II trial were announced in September. AZT and Burroughs Wellcome's ganciclovir (DHPG) were given orphan product designations last year. Wellferon, in Phase III clinicals for Kaposi's Sarcoma, is also in clinicals for the treatment for two types of benign tumors, laryngeal growths in children and genital warts in adults. Ganciclovir has an orphan indication for treatment of severe human cytomegalovirus infections in specific immuno-suppressed patient populations (e.g., bone marrow transplant recipients and AIDS). Burroughs Wellcome notes that the gangciclovir is being studied only for treatment of severe CMV infections because it "has a narrow therapeutic index and apparently causes a reduction in white blood cells as well as testicular damage in animals when given in moderate to high doses." Warner Lambert reported results of studies with its AIDS drug trimetrexate at a recent meeting with financial analysts ("The Pink Sheet" Nov. 10, p. 10). The firm said trimetrexate administered in combination with leucovorin achieved a 70% success rate in AIDS patients with pneumocystis carinii. Merieux said it has almost completed an initial pilot study of Imuthiol involving 40 patients at M.D. Anderson Hospital. A dose-response trial is set to begin in Tucson, Arizona and a multi-center trial is expected to begin at the end of November. Merrell Dow's eflornithine has two orphan indications, pneumocystis carinii and sleeping sickness. The company announced in May that 49 of 66 patients with pneumocystis carinii were cured of the pneumonia after a 14-day treatment cycle with eflornithine. The drug is in Phase III clinicals for pneumonia and is also under investigation for treatment of metastatic malignant melanoma. The four AIDS treatment products are included among seven biologicals and 19 drugs in a FDA tabulation of orphan designations made between January and September. On the biologics side, Xoma'santi T lymphocyte immunotoxin XMMLY-H65-RTA was granted orphan status for ex vivo treatment to eliminate mature T cells from potential bone marrow grafts and for in vivo treatment of bone marrow recipients to prevent graft rejection and graft vs. host disease. It is the firm's fourth orphan monoclonal antibody. Last year, FDA designated three Xoma monoclonals as orphans, one for the diagnosis and one for the treatment of melanoma, and one for treatment of progressed gram-negative sepsis. Damon Biotech also obtained an orphan indication for its monoclonal antibodies for the treatment of B-cell lymphoma. Among other biologicals, Amgen received an orphan designation for recombinant human erythropoietin for the treatment of anemia associated with end stage renal disease. The company began U.S. clinicals with recombinant erythropoietin last December ("The Pink Sheet" Feb. 17, p. 13). On Nov. 17, FDA also announced that it has awarded 21 grants ($2.8 mil.) for orphan product development. The grants will help finance development of: histrelin (LHRH analog) for prevention of "rare life threatening attacks of the nervous system which may occur during the menstrual cycle; epidural clonidine for postoperative pain control; intrathecal baclofen for severe spasticity; and dexamethasone to prevent bronchopulmonary dysplasia. Chart omitted.

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