The U.S. Food and Drug Administration approved 13 new cancer drugs in 2012, three of them via the accelerated approval process.

The year 2012 continued the upward trend in oncology drug approvals issued by the U.S. Food and Drug Administration (FDA). The Office of Hematology and Oncology Products (OHOP), in the FDA's Center for Drug Evaluation and Research, gave the green light to 13 new agents—up from 11 in 2011 and just 2 in 2010.

OHOP's director, Richard Pazdur, MD, says the increase reflects better science in drug development. While cytotoxic agents were previously subjected to what Pazdur describes as a “roulette wheel of phase II trial testing,” looking for cancers in which the treatments might work, most of the newly approved drugs are directed at specific targets in cancer cells.

Additionally, Pazdur says, ascertaining drug efficacy with these personalized treatments is easier for FDA reviewers. “What we've seen in the past 2 years is that more sophisticated approaches are coming to fruition,” he says.

Newly approved Iclusig (ponatinib), for instance, marketed by ARIAD Pharmaceuticals of Cambridge, MA, targets chronic myeloid leukemia (CML) cells with a T315I gene mutation. Patients with CML have few other treatment options, so ponatinib was among the 3 drugs reviewed and given a go-ahead under the FDA's accelerated approval process.

Accelerated approval decisions were generally based on surrogate endpoints—such as normalization of white cell counts in leukemia—rather than the survival benefits anticipated in randomized confirmation trials that will follow, says Pazdur.

Christopher-Paul Milne, DVM, director of research at the Tufts Center for the Study of Drug Development in Boston, MA, cautions that the viability of surrogate endpoints remains an open question about the accelerated approval process. “We may have to wait a few years to see if they translate to survival benefits,” he says. “Confirmatory testing in accelerated approval is a long-term process.”

Here is the list of approved drugs:

  • Bosulif (bosutinib; Pfizer), for CML

  • Cometriq (cabozantinib; Exelixis), for metastatic medullary thyroid cancer

  • Erivedge (vismodegib; Genentech), for basal cell carcinoma

  • Iclusig (ponatinib; ARIAD Pharmaceuticals), for CML and Philadelphia chromosome–positive acute lymphoblastic leukemia

  • Inlyta (axitinib; Pfizer), for advanced renal cell carcinoma

  • Kyprolis (carfilzomib; Onyx Pharmaceuticals), for previously treated multiple myeloma

  • Neutroval (tbo-filgrastim, Teva Pharmaceutical Industries), for reducing the length of time during which certain patients receiving cancer chemotherapy experience severe neutropenia

  • Perjeta (pertuzumab; Genentech), for metastatic HER2-positive breast cancer

  • Stivarga (regorafenib; Bayer Health-Care and Onyx Pharmaceuticals), for metastatic colorectal cancer

  • Synribo (omacetaxine mepesuccinate; Teva Pharmaceutical Industries), for CML

  • Voraxaze (glucarpidase; BTG International), for toxic blood levels of methotrexate due to kidney failure

  • Xtandi (enzalutamide; Astellas Pharma/Medivation), for metastatic castration-resistant prostate cancer

  • Zaltrap (ziv-aflibercept; Sanofi and Regeneron Pharmaceuticals), in combination with chemotherapy, for colorectal cancer.

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