FDA Expands Use of Ibrutinib, Fast Track Designation Granted to SGX301, and more

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 used in Europe since 2008 to help cancer patients boost their neutrophil counts while undergoing chemotherapy, but this would be the first biosimilar drug approved in the United States. The FDA panel endorsed the new drug after finding “no clinically meaningful differences between the proposed product and the reference product in term of safety, purity, and potency.” Unlike traditional generics, biosimilar drugs are “highly similar” versions of complex biologic drugs, which are produced from living cells and more expensive and harder to manufacture than ordinary chemical drugs. This approval could pave the way for lower-cost versions of expensive drugs in the future.
Source: The Wall Street Journal, FDA Oncologic Drugs Advisory Committee

CPX-351 Receives Fast Track Designation for AML in Elderly Patients
The FDA recently granted Fast Track designation to CPX-351 for the treatment of elderly patients with secondary acute myeloid leukemia (AML). The experimental drug is a liposomal formulation of the chemotherapeutic agents cytarabine and daunorubicin that is intended to deliver synergistic drug ratios to leukemia cells. In a randomized phase 2 study presented last year, CPX-351 induced greater response rates in patients with newly-diagnosed AML, compared to conventional administration of the two agents (66.7% vs. 51.2%; p=0.07). Another analysis demonstrated that CPX-351 led to higher response rates (57.6% vs. 31.6%; p=0.06), longer event-free survival (HR=0.59; p=0.08), and longer overall survival (HR=0.46; p=0.01) in patients with secondary AML. A phase 3 trial designed to evaluate CPX-351 in patients with newly diagnosed secondary AML is currently underway.

Source: Celator Pharmaceuticals press release

Anti-Clotting Drug Edoxaban Receives FDA Approval
Edoxaban, a factor Xa inhibitor, was recently approved to reduce the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation. Edoxaban also has been approved to treat deep vein thrombosis (DVT) and pulmonary embolism (PE) in patients previously treated with an anti-clotting drug administered by injection or infusion (parenterally), for five to 10 days. The FDA’s approval was based on results from a clinical trial of 21,105 patients which demonstrated that a higher dose of edoxaban (60 mg) was as effective as warfarin in reducing the risk of stroke; in addition, edoxaban had significantly fewer major bleeding events compared to warfarin. Edoxaban was also as effective as warfarin in reducing the rate of recurrent VTE in patients with a DVT and/or PE (3.2% with edoxaban vs. 3.5% with warfarin). The most commonly observed side effects in these trials were bleeding and anemia. “In patients with atrial fibrillation, anti-clotting drugs lower the risk of stroke by helping to prevent blood clots from forming in the heart,” said Norman Stockbridge, MD, PhD, director of the Division of Cardiovascular and Renal Products in the FDA’s Center for Drug Evaluation and Research. “It is important to have a variety of these types of drugs available as options for patients.”
Source: FDA.gov

IOM Recommends Wider Sharing of Clinical Trial Data
In a new 249-page report, the Institute of Medicine (IOM) urges government agencies and pharmaceutical companies to share data from IOM-funded research studies.
The report outlines several steps that clinical trial sponsors should undertake to improve access, including developing specific plans for sharing data (i.e., granting access through third-party websites) and adhering to recommended timetables for releasing both summary and complete data packages. Specifically, the report suggests, summary level results, including adverse event information, should be made publicly available no later than one year after a trial has been completed; a complete data package should be shared no later than 18 months after a study is completed.

“The issue is no longer whether to share clinical trial data, but what specific data to share, at what time, and under what conditions,” said Bernard Lo, MD, who chaired the IOM committee that generated the report. “Our recommendations represent an attempt to balance the interests of different stakeholders with the public interest of having the best information possible regarding the effectiveness and safety of therapies.” ●

Source: The Institute of Medicine’s “Sharing Clinical Trial Data” report