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FDA Actions & Updates

The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.

Wednesday, November 19, 2014

The U.S. Food and Drug Administration has granted orphan drug designation to BGB324 for the treatment of patients with acute myeloid leukemia (AML). BGB324 is a selective inhibitor of the Axl receptor tyrosine kinase, which blocks epithelial-mesenchymal transition, a key driver in drug-resistance and metastasis.


The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


Approximately 18,860 new cases of AML were diagnosed in the U.S. in 2014, and approximately 10,460 individuals died as a result of the disease, according to American Cancer Society estimates.


BGB324 is currently being evaluated in a multicenter Phase Ib trial for patients with AML as both a single agent and in combination with cytarabine (the current standard of care drug for patients with AML). Endpoints for the trial include safety, tolerability, clinical response, and assessment of novel biomarkers. Data are expected from the trial in 2015, and additional trials with the drug in non-small cell, lung cancer are in preparation, according to a news release from the drug’s manufacturer, BerGenBio AS.

Monday, November 17, 2014

The U.S. Food and Drug Administration has approved Avastin (bevacizumab) in combination with chemotherapy for the treatment of patients with platinum-resistant, recurrent, epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received no more than two prior chemotherapy regimens. Avastin is a biologic antibody that binds to vascular endothelial growth factor (VEGF) to prevent tumor metastasis by inhibiting tumor blood supply. Avastin is indicated with paclitaxel, pegylated liposomal doxorubicin or topotecan chemotherapy.


Avastin was granted priority review for this indication and for persistent, recurrent, or metastatic cervical cancers earlier this year (OT 9/10/14 issue). And, the drug also has approved indications for the treatment of patients with metastatic colorectal cancer (OT 2/25/13 issue); and for the treatment of patients with first-line or previously untreated metastatic colorectal cancer (OT 3/25/04 issue).


The approval for the new indication was based on data from the multicenter, randomized, Phase III AURELIA study of 361 women with platinum-resistant, recurrent, epithelial ovarian, primary peritoneal, or fallopian tube cancer, who had received no more than two anticancer regimens prior to enrollment in the trial. Patients were randomized to one of six treatment arms (paclitaxel, topotecan or pegylated liposomal doxorubicin with or without Avastin). Median progression-free survival was 6.8 months for women receiving Avastin plus chemotherapy, compared with 3.4 months for women receiving chemotherapy alone.


Adverse events were consistent with those seen in previous trials of Avastin across tumor types for approved indications, but also included high blood pressure and pain, redness or swelling of the hands or feet.


Avastin is marketed by Genentech.   

Tuesday, November 11, 2014

The U.S. Food and Drug Administration has granted Qualified Infectious Disease Product (QIDP) designation to the oral drug RHB-105 as first line treatment for patients with Helicobacter pylori (H. pylori) infection regardless of ulcer status. H. pylori is a known cause of gastric cancer and mucosa associated lymphoid tissue (MALT) lymphoma, as well as chronic gastritis and peptic ulcer disease.


This indication for RHB-105 is significantly broader than current standard treatments for H. pylori, which are typically indicated to treat patients with active or recent history of ulcers, according to a news release from the drug manufacturer, RedHill Biopharma Ltd.


The FDA’s QDIP designation, established under the Generating Antibiotic Incentives Now (GAIN) Act, is intended to encourage the development of new antibiotic drugs for the treatment of serious or life-threatening infections. The designation also gives the drug Fast-Track development status, Priority Review of a potential future New Drug Application, and an additional five years of exclusivity for the product, if the drug is approved.


H. pylori was recently added by the FDA to the Agency’s list of qualifying pathogens that have the potential to pose a serious threat to public health, which was also established under the GAIN Act. In addition to H. pylori increasing risk of ulcers and other gastric diseases, research suggests between 35 and 60 percent of gastric adenocarcinomas are attributable to H. pylori infection; and H. pylori is estimated to be responsible for approximately two-thirds of gastric lymphomas (Clinical and Translational Gastroenterology, 2013;4:e32).


RHB-105, a fixed-dose combination therapy of two antibiotics and a proton pump inhibitor, is currently undergoing Phase III clinical testing in the U.S. Data from phase II studies in Australia showed the drug had eradication rates that exceeded 90 percent.

Wednesday, November 05, 2014

The U.S. Food and Drug Administration has approved the use of Cyramza (ramucirumab) in combination with paclitaxel to treat patients with advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose cancer has progressed after treatment with fluoropyrimidine- or platinum-containing chemotherapy.


Cyramza—an angiogenesis inhibitor that blocks the blood supply to tumors—is now the only FDA-approved second-line treatment option for patients with advanced or metastatic gastric or GEJ adenocarcinoma whose disease has progressed on or after prior chemotherapy, notes a news release from the manufacturer, Eli Lily.


The drug has also been granted Orphan Drug Designation by the FDA for this indication. Orphan drug status is given in the U.S. by the FDA's Office of Orphan Products Development to medicines that show promise for the treatment of rare diseases. Cyramza was approved first approved earlier this year for the treatment of patients with advanced stomach cancer or GEJ adenocarcinoma (OT 5/25/14 issue).


The approval for this indication for Cyramza is based on data from the Phase III RAINBOW trial, which included 665 patients from 27 countries with locally advanced or metastatic gastric or GEJ adenocarcinoma whose cancer had progressed after fluoropyrimidine- and platinum-containing chemotherapy. The patients were randomized to receive Cyramza plus paclitaxel or placebo plus paclitaxel. Median overall survival for patients receiving the Cyramza combination was 9.6 months compared with 7.4 months for those receiving placebo plus paclitaxel. Progression-free survival for patients receiving Cyramza was 4.4 months, compared with 2.9 months for those receiving the placebo.


The most common adverse events for patients who received Cyramza in the trial were fatigue, neutropenia, diarrhea, and epistaxis. The most common serious events were neutropenia and febrile neutropenia. And, 19 percent of patients treated with Cyramza received granulocyte colony-stimulating factors as treatment for low white blood cells.


The labeling for Cyramza contains a Boxed Warning for hemorrhage and additional Warnings and Precautions for arterial thromboembolic events, hypertension, infusion-related reactions, gastrointestinal perforations, impaired wound healing, clinical deterioration in patients with Child-Pugh B or C cirrhosis, and reversible posterior leukoencephalopathy syndrome.

Monday, October 27, 2014

The U.S. Food and Drug Administration has today granted Breakthrough Therapy Designation to Keytruda (pembrolizumab), an anti-PD-1 therapy, for the treatment of patients with non-small cell lung cancer  that has progressed on or following platinum-based chemotherapy and that is also epidermal growth factor receptor (EGFR) mutation-negative and anaplastic lymphoma kinase (ALK) rearrangement-negative.


The breakthrough therapy designation, enacted as part of the FDA’s 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.


Keytruda was recently approved for the treatment of patients with advanced or unresectable melanoma who are no longer responding to other drugs under the FDA’s accelerated approval program (OT 10/10/14 issue). Keytruda also received breakthrough therapy designation, priority review, and orphan drug status for that indication in melanoma.


The Phase 1b KEYNOTE-001 study of Keytruda in advanced non-small cell lung cancer is currently ongoing. Updated findings from that trial were reported at the recent European Society of Medical Oncology 2014 Congress.


Keytruda is marketed by Merck.

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