FDA Approves Drug for Rare Cancer

The FDA has approved Istodax (romidepsin), an injectable medication to treat patients with cutaneous T-cell lymphoma (CTCL), a rare form of cancer.
Cutaneous T-cell lymphoma is a slow-growing cancer of T-lymphocytes, infection-fighting white blood cells. Most cases start with dry skin, red rash and itching that can become severe. The skin may develop tumors that can become ulcerated, causing infection. In some cases, CTCL spreads to the blood, lymph nodes or internal organs. Patients with localized CTCL on the skin are treated with topical agents or phototherapy, but chemotherapy may be used if the cancer advances. About 1,500 new cases of CTCL are diagnosed every year in the U.S.

Istodax, marketed by Gloucester Pharmaceuticals, interferes with processes required for cell replication. It is intended to be used by patients when CTCL gets worse or comes back after at least one other type of chemotherapy has been used.

"This approval demonstrates [the] FDA's commitment to the development and approval of drugs for rare and uncommon diseases," said Richard Pazdur, MD, director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research.

Previous approvals for CTCL included Zolinza (vorinostat), Ontak (denileukin difitox) and Targretin (bexarotene).

Istodax was evaluated based on two clinical studies involving a total of 167 patients. About 35 percent of patients in both trials experienced tumor responses, indicating a reduction of the size of tumors. Responses lasted a median of 15 months in one study and 11 months in the other study. Six percent of those participants studied had complete responses, indicating no apparent evidence of the tumor on physical, laboratory and X-ray examinations.

Common side effects of Istodax include nausea, fatigue, infections, vomiting, decreased appetite, decreased red blood cell count, decreased platelet count and decreases in the components of white blood cells.

Istodax may cause changes in an electrocardiogram (ECG). Periodic blood tests should be done to monitor electrolytes, and periodic ECG monitoring should be considered in patients at risk for certain heart rhythm abnormalities. Istodax may harm a fetus, so women should not become pregnant while taking the drug.

Contact:
Karen Riley
301-796-4674
karen.riley@fda.hhs.gov