FDA Approves Bendamustine, Novel Agent for Chronic Lymphocytic Leukemia

Cephalon recently announced that the US Food and Drug Administration (FDA) has approved bendamustine hydrochloride (Treanda) for the treatment of patients with chronic lymphocytic leukemia (CLL). The American Cancer Society estimates that more than 15,000 new cases of this rare disease will be diagnosed in the United States this year. The bendamustine application as a CLL treatment received priority review from the FDA and was approved within 6 months of the September 2007 submission. Cephalon anticipates that bendamustine will be available to physicians and patients as a CLL treatment in the United States in April 2008.

Effective New Option
“Treanda is an important new treatment for patients with chronic lymphocytic leukemia, and this first-cycle approval by FDA represents a significant milestone in the growth of our oncology business,” said Dr. Lesley Russell, executive vice president, Worldwide Medical and Regulatory Operations. “With a strong pipeline of near- and longer-term opportunities, Cephalon Oncology is poised to deliver therapies that target both hematologic cancers and solid tumors for patients in need of new options.”

Dr. Bruce Cheson, clinical professor of hematology/oncology, Georgetown University School of Medicine, stated, “Patients with chronic lymphocytic leukemia can often live normal lives for many years because of treatments that control the disease over the long-term. Treanda is an effective new option that offers a delay in disease progression, an important goal for patients with chronic lymphocytic leukemia.”

Pivotal Study
In a randomized, international, multicenter, open-label pivotal study of 301 treatment-naive patients with CLL, those who received bendamustine had better clinical outcomes compared to patients treated with chlorambucil (Leukeran), an FDA-approved chemotherapy for patients with CLL. Specifically, bendamustine recipients had a significantly higher overall response (59% of patients responded to bendamustine vs 26% to chlorambucil; P < .0001). Patients who received bendamustine also had a higher complete response rate than those treated with chlorambucil (8% vs < 1%), which means that after treatment with bendamustine, some patients had no signs of disease in their blood.

Patients receiving bendamustine also had a significantly longer progression-free survival (18 vs 6 months; hazard ratio = 0.27; P < .0001). The response to bendamustine lasted longer than the response in patients who received chlorambucil (19 vs 7 months). The most common adverse events in the trial were myelosuppression, fever, nausea, and vomiting.

Bendamustine has been granted orphan drug status by the FDA for the treatment of CLL. The orphan drug designation will provide marketing exclusivity in this indication until March 2015.