FDA Oncology Updates

•Under an accelerated approval process, the FDA approved Arzerra (ofatumumab) by GlaxoSmithKline (GSK) for patients with chronic lymphocytic leukemia refractory to other drugs. GSK will need to complete additional study of the drug to demonstrate that adding Arzerra to standard chemotherapy delays disease progression.

•The FDA declined to approve Amgen’s Prolia (denosumab) in breast and prostate cancer patients with bone loss associated with hormone ablation therapy pending data from additional trials that show Prolia does not negatively affect time-to-disease progression or overall survival. Amgen said it expects to have data available in the first quarter of 2010.

• Merck received an expanded indication from the FDA for its HPV vaccine Gardasil, now approved for males aged 9 to 26 years. The CDC’s immunization advisory panel declined to classify the vaccine as “recommended” for males, however, saying it was not cost-effective.

•In September, the FDA sent a letter warning Baxter International that its marketing of the Isolex cancer device was “false and misleading” and that the company had failed to meet federal manufacturing standards in producing the device. Baxter said it is working to address the FDAs concerns.

•The FDA approved GSK’s Votrient (pazopanib) for advanced renal cell carcinoma despite concerns from its staff reviewers about possible liver toxicity. The ODAC panel unanimously recommended approval based on improvement in progression-free survival. Two hematologists on the panel said the association between Votrient and the patients’ liver damage was inconclusive.

•Elitek (rasburicase) by Sanofi-aventis was recently approved by the FDA in the initial management of plasma uric acid (PUA) levels in adults with leukemia, lymphoma, and solid tumors who are receiving anticancer therapies known to cause tumor lysis syndrome and subsequent elevations of PUA.

•Istodax (romidespin) has been approved by the FDA to treat cutaneous T-cell lymphoma. Istodax is a new injectable medication, and 35% of patients in two clinical studies demonstrated tumor responses lasting a median of 11 to 15 months. In addition, 6% had complete response.

•Cequent Pharmaceuticals announced that it has filed an Investigational New Drug application with the FDA for CEQ508, an oral RNA interference drug. The company is preparing to conduct a phase I trial that will enroll 18 patients with familial adenomatous polyposis, a gastrointestinal disease that generally leads to colorectal cancer.

•The FDA sent a Complete Response Letter to GTX Inc, a company in Memphis, Tennessee, that is developing toremifene citrate, an experimental prostate cancer drug. The FDA said a second phase III trial is needed to demonstrate that the drug effectively reduces fractures in men receiving androgen blockers without negatively affecting time-to-disease progression or overall survival. GTx has requested a meeting with the FDA to discuss the request.

•Dendreon has completed the paperwork for its Biologics License Application on behalf of Provenge, a vaccine that has been demonstrated to improve outcomes for men with prostate cancer. The Seattle-based company is anticipating FDA approval for the immunotherapy agent and a subsequent launch in 2010.

•Abraxis has been granted orphan drug status for Nab-paclitaxel, its proprietary albumin-bound paclitaxel agent approved for breast cancer, for stage IIB-IV melanoma. The drug was previously granted orphan drug status in September for pancreatic cancer. Abraxis will test the drug in a phase III study against dacarbazine in patients with chemotherapy-naïve melanoma.

•GenVec is another company that has been granted orphan drug status from the FDA, for its experimental treatment TNFerade, which is being investigated for multiple indications. Interim data from a phase III trial of TNFerade in patients with advanced pancreatic cancer were promising, and the orphan drug status is based on these results. The drug is injected directly into the tumor site, where it stimulates an immune response that ultimately causes apoptosis and tumor shrinkage.

•NovaRx Corporation has received FDA approval for a Special Protocol Assessment Protocol Amendment for a phase III trial of its drug Lucanix, which is being investigated in patients with non-small cell lung cancer. The amendment allows patients with stable brain metastases to participate in the trial, which the company says will make it easier to enroll more patients. Other changes were also made that expand patient eligibility.

•Yet another drug received an orphan drug designation from the FDA in November. Voreloxin, a treatment for acute myeloid leukemia (AML) by Sunesis Pharmaceuticals, will be investigated in two phase II clinical trials in elderly patients with newly diagnosed AML who are unlikely to respond to standard chemotherapy. A third study will look at the novel drug in combination with cytarabine.