ASH Clinical News February 2015 | Page 17

CLINICAL NEWS Latest & Greatest FDA Expands Use of Ibrutinib for Waldenström Macroglobulinemia The U.S. Food and Drug Administration (FDA) approved the expanded use of ibrutinib to treat Waldenström macroglobulinemia (WM), a rare hematologic malignancy. The drug received a “breakthrough therapy” designation for this use, as well as an orphan product designation. Approval of the new indication was based on results from a clinical study of 63 previously treated WM patients. Ibrutinib was orally administered to all participants in a daily 420 mg dose until disease progression or side effects became intolerable. Sixty-two percent of participants had their cancer shrink after treatment, and the duration of response ranged from 2.8 months to approximately 18.8 months. The most common side effects associated with the drug are thrombocytopenia, neutropenia, diarrhea, and anemia. The new WM approval marks the fourth indication for ibrutinib; it is also approved for mantle cell lymphoma, previously treated chronic lymphocytic leukemia (CLL), and del(17p) CLL. Source: FDA.gov FDA Grants Fast Track Designation to SGX301 SGX301 recently received fast track designation as first-line treatment of cutaneous T-cell lymphoma (CTCL); the drug previously received orphan drug designation from the FDA. CTCL constitutes a rare group of non-Hodgkin lymphomas (NHLs), occurring in about 4 percent of the approximately 50,000 individuals diagnosed with NHL each year. A protocol for a pivotal, double-blind, randomized, placebo-controlled, multicenter trial of approximately 120 patients has also been cleared through the FDA. Source: Solgenix press release FDA Panel Recommends First “Biosimilar” Drug The FDA’s Oncologic Drugs Advisory Committee unanimously recommended the agency approve the drug known as Zarzio®, a “biosimilar” version of Amgen’s hematopoietic growth factor Neupogen® (filgrastim). Zarzio and two other biosimilar drugs like Neupogen have been \