Green light based on mid-stage results.
Patients with multiple myeloma and no options have new hope with the approval of Onyx Pharmaceuticals’ Kyprolis. The U.S. Food and Drug Administration has approved Kyprolis, also known as carfilzomib, to treat patients with the cancer who have received at least two prior therapies, including treatment with Velcade (bortezomib) and an immunomodulatory therapy.
“The approval of Kyprolis provides a treatment option to patients with multiple myeloma whose disease has progressed despite use of available therapies,” says Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. “We are encouraged by the continued progress in the development of drugs for multiple myeloma over the past decade, offering improved treatment of this disease.”
While the approval is limited to patients for whom there is no other option, it is a big win for Onyx, which won approval on mid-stage data from a single-arm study. It is also a big win for the investors of Proteolix, the developer of carfilzomib, which was acquired by Onyx in 2009 for $276 million plus up to $575 million in milestones, including $170 million for accelerated approval.
The approval followed an advisory committee vote for approval of the drug last month. It decided that the benefits for the limited group of patients outweighed the risks, and it was worth approving the proteasome inhibitor right away rather than waiting for confirmatory results from a late stage trial in progress.
Multiple myeloma is a form of blood cancer that arises from plasma cells, usually growing in bone marrow, the place where normal blood cells are produced. In 2012, nearly 22,000 people will be diagnosed with multiple myeloma and more than 10,000 will die from the disease, according to the American Cancer Society.
Approval of Kyprolis, which is administered intravenously, was based on results from a study of 266 patients with relapsed multiple myeloma who had received at least two prior therapies, including Velcade and Thalomid (thalidomide).
The study was designed to measure the percentage of patients who experienced complete or partial disappearance of tumor after treatment, the overall response rate. The overall response rate was 23 percent. The median duration of response was 7.8 months.
Kyprolis is being approved under the FDA's accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. The program is designed to provide patients with earlier access to promising new drugs. The company is still required to submit additional clinical information after approval to confirm the drug's clinical benefit.
By MARIE DAGHLIAN