From an immunotherapy that improves melanoma survival to a device that zaps brain tumors, 2011 was a year of firsts from the FDA. This is our second annual guide to approvals of new cancer drugs, indications, and devices – along with a review of safety warnings and other actions affecting oncology practices.
(Click here for a PDF version of The Oncology Report Guide to Cancer Drugs and Devices in 2011.)
NEW DRUG APPROVALS
• Abiraterone acetate (Zytiga Tablets, Centocor Ortho Biotech, Inc.). An androgen suppressant that decreases testosterone production for use in combination with prednisone for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) who have received prior chemotherapy containing docetaxel. The fourth approval in late-stage prostate cancer since April 2010.
Basis: An international study of 1,195 men who had previously received chemotherapy containing docetaxel. Median overall survival was 14.8 months with abiraterone plus prednisone, vs. 10.9 months with prednisone alone.
© Aydin Mutlu/iStockphoto.com
This guide covers all the major drug approvals, new devices and label changes in the oncology world for 2011.
• Asparaginase Erwinia chrysanthemi [Erwinaze for injection, EUSA Pharma (USA), Inc.] An asparagine-specific enzyme approved as a component of a multiagent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL) who developed hypersensitivity to E. coli-derived asparaginase. An orphan drug, it works by the same mechanism as the two previously approved treatments that block asparagine, a protein necessary for the proliferation of neoplastic cells.
Basis: In a single-arm, multicenter, open-label study of 58 patients, all 48 patients with available samples achieved threshold trough asparaginase levels shown to correlate with asparagine depletion and with serum levels that predict clinical efficacy.
• Brentuximab vedotin (Adcetris for injection, Seattle Genetics, Inc.). A CD30-directed antibody-drug conjugate for the treatment of Hodgkin’s lymphoma after failure of autologous stem cell transplant (ASCT) or at least two prior multiagent chemotherapy regimens in patients who are not ASCT candidates, and for the treatment of systemic anaplastic large cell lymphoma (ALCL) after failure of at least one prior multiagent chemotherapy regimen. The first new treatment approved for Hodgkin’s since 1977 and the first treatment approved for ALCL.
Basis: In a single-arm study of 102 patients treated with brentuximab, 73% had either a complete or partial response. In a similarly designed study of 58 patients with ALCL, 86% achieved a partial or complete response.
Addendum: An accelerated approval, based on clinical data suggesting that treatment resulted in significant responses; conversion to full approval is contingent on confirmation of benefits in follow-up studies. (In January 2012, the FDA added a warning about the risk of a rare brain infection to the label.)
• Crizotinib (Xalkori Capsules, Pfizer, Inc.). A kinase inhibitor for the treatment of locally advanced or metastatic non–small cell lung cancer (NSCLC) that express an abnormal anaplastic lymphoma kinase (ALK) gene, as detected by the Vysis ALK Break-Apart FISH Probe Kit (Abbott Molecular, Inc.), a companion diagnostic test approved at the same time.
Basis: In two single-arm studies of 255 patients with locally advanced or metastatic, ALK-positive NSCLC, most of whom had received prior chemotherapy, the overall response rate was 50% and 61%, with a median response duration of 42 and 48 weeks, respectively.
Addendum: An accelerated approval, so conversion to full approval is contingent on follow-up studies confirming clinical benefit.
• Deferiprone (Ferriprox Tablets, ApoPharma, Inc.). An oral iron chelator for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.
Basis: A prospective pooled analysis of previously conducted studies of 236 patients (most had thalassemia syndromes) with transfusion-dependent iron overload, for which previous iron chelation therapy had failed or was considered inadequate because of poor tolerance. Within 1 year of starting treatment, 50% of the patients had at least a 20% decrease in serum ferritin levels.
Addendum: An accelerated approval. Safety and effectiveness not established for the treatment of transfusional iron overload due to other chronic anemias, including sickle cell disease. As a condition of approval, ApoPharma will conduct a study of safety and efficacy in patients with sickle cell disease and transfusional iron overload not adequately treated with available chelating agents.
• Hemacord (New York Blood Center). A hematopoietic progenitor cells–cord (HPC-C) preparation for allogenic hematopoietic stem cell transplantation. The first approval of a license for an umbilical cord blood therapy.
Basis: Safety and effectiveness data submitted to a public docket and data submitted in the license application demonstrating compliance with other regulatory requirements.
• Ipilimumab injection (Yervoy, Bristol-Myers Squibb). A human cytotoxic T-lymphocyte antigen (CTLA-4)-blocking antibody for the treatment of unresectable or metastatic melanoma. The first treatment proven to prolong survival in patients with metastatic melanoma.