FDA approves denosumab for giant cell tumors


Denosumab is approved for the treatment of giant cell tumors of the bone, the Food and Drug Administration announced June 13.

Rare and usually noncancerous, giant cell tumors of the bone generally affect 20- to 40-year-olds. Most of these tumors destroy growing bone, causing pain, limited range of motion, and bone fractures. Rarely, the tumors become cancerous and spread to the lungs.

Denosumab (Xgeva) is indicated for use in patients who are not candidates for surgical resection of their tumors or when surgery is likely to result in severe morbidity, such as loss of limbs or joint removal. It should be used only in adolescents whose bones have matured, the FDA said in a statement.

"Today’s approval of Xgeva provides a needed treatment option for patients with GCTB who are not surgical candidates or who would otherwise have to undergo extensive, life-altering surgery," said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

The FDA reviewed denosumab under its priority review program as the drug was granted orphan product designation because GCTB is a rare disease.

The safety and effectiveness of denosumab for GCTB were established in two clinical trials that enrolled 305 adult or adolescent patients with confirmed GCTB that was recurrent, unresectable, or would be associated with severe morbidity if surgically managed.

After an average of 3 months, tumors reduced in size among 47 of 187 patients whose tumors could be measured. Over an average follow-up of 20 months, GCTBs regrew in three patients whose tumors originally became smaller during treatment.

Common side effects of denosumab included joint pain, headache, nausea, fatigue, back pain, and extremity pain. The most common serious side effects were osteonecrosis of the jaw and osteomyelitis. Women of reproductive potential should use highly effective contraception while taking denosumab because of potential fetal harm, according to the FDA.

The recommended dose and schedule of denosumab for the treatment of giant cell tumor of bone is 120 mg administered subcutaneously every 4 weeks with additional 120 mg doses on days 8 and 15 of the first month. Full prescribing information is available.

Denosumab was approved in 2010 to prevent fractures when cancer has spread to the bones. It is marketed by Amgen.

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