Carfilzomib Receives FDA Nod for Multiple Myeloma

Nick Mulcahy


July 20, 2012

July 20, 2012 — The US Food and Drug Administration (FDA) has approved carfilzomib (Kyprolis, Onyx Pharmaceuticals) today for the treatment of multiple myeloma in patients who have received at least 2 prior therapies, including bortezomib (Velcade, Millennium Pharmaceuticals) and an immunomodulatory therapy.

The new intravenous drug has been given the green light under the FDA's accelerated approval program, which requires the company to eventually submit additional data to confirm clinical benefit.

"The approval of Kyprolis provides a treatment option to patients with multiple myeloma whose disease has progressed despite use of available therapies," said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research, in an FDA news release. "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."

The safety and effectiveness of carfilzomib were evaluated in a study of 266 patients with relapsed multiple myeloma who had received at least 2 prior therapies, including bortezomib and thalidomide.

The study was designed to measure complete or partial response to treatment or the overall response rate. The overall response rate was 23%. The median duration of response was 7.8 months.

The FDA's Oncologic Drugs Advisory Committee voted unanimously in June to recommend the accelerated approval of carfilzomib, as reported by Medscape Medical News.

One committee member suggested that unmet patient need was an important factor in the decision-making.

Patients with end-stage multiple myeloma who have been heavily pretreated have few, if any, viable options, said advisory panel member Mikkael Sekeres, MD, at the time. He is associate professor of medicine at the Cleveland Clinic Taussig Cancer Institute in Ohio. "Today, we voted to make one option available to them. The response rate was acceptable for this population, and safety was also acceptable," he said.

After the committee's vote, another expert called carfilzomib "potentially important" but emphasized its unknowns. On his Medscape videoblog, Bruce D. Cheson, MD, from Georgetown University in Washington, DC, said: "Will it be used, and how will it be used? That remains to be seen. Will it be moved up front because it has less neurotoxicity than bortezomib? I don't know. We do need active drugs with less toxicity."

Dr. Cheson suggested that clinicians should "decide on your own level of enthusiasm for carfilzomib."

In the pivotal study, the most common adverse effects (observed in more than 30% of the study participants) for carfilzomib were fatigue, anemia, thrombocytopenia, shortness of breath, diarrhea, and fever. Serious adverse effects seen with carfilzomib included heart failure and shortness of breath. Patients should be monitored closely and treatment withheld if these serious adverse effects occur, according to the FDA.

In 2012, an estimated 21,700 people will be diagnosed with multiple myeloma and 10,710 will die from the disease, according to the American Cancer Society.


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