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Biotech Tracker: Thalomid: What’s in a Label?
February 23rd 2004. Celgene (NASDAQ: CELG) announced that the FDA has accepted for review its supplemental NDA for Thalomid (thalidomide) for the treatment of multiple myeloma. Thalidomide, infamous because of the severe birth defects it caused when given to pregnant women, has a wide variety of therapeutic uses. Since it is currently approved in the U.S. for the treatment of a specific complication of leprosy, thalidomide can be, and has been, used off-label in cancer. In fact, it is prescribed far more often for cancer than for leprosy. However, Celgene cannot promote use for cancer to doctors.

Thalomid’s adverse effect profile continues to be reassuring, given that physicians know to avoid using thalidomide during pregnancy. In spite of delays in gaining approval for the treatment of multiple myeloma, we expect that Thalomid will become an important part of the management of several indications. In addition to multiple myeloma, Thalomid is being tested in late-stage trials relating to non-specific immune disorders, apthous ulcers, and AIDS wasting syndrome. Thalomid is in mid- and late-stage programs in HIV-related diarrhea, AIDS-related Kaposi sarcoma, leukemia, and various solid tumors. Thalomid is currently used off-label in Crohn disease, and has generated encouraging mid-phase data in the management of Waldenström macroglobulinemia, myelodysplastic syndrome, metastatic melanoma, and inflammatory bowel disease. The jury is still out on Thalomid’s ability to treat conditions such as refractory pediatric malignancies and metastatic renal (kidney) cell carcinoma.

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