FDA approves Onyx Pharmaceuticals' Kyprolis for advanced multiple myeloma

Onyx Pharmaceuticals announced Friday that the FDA granted accelerated approval for the injectable drug Kyprolis (carfilzomib) to treat patients with multiple myeloma whose disease progressed despite treatment with at least two prior therapies, including treatment with Velcade (bortezomib) and an immunomodulatory therapy. Onyx shares climbed as much as 13 percent on the news.

The approval was based on the results of the Phase IIb 003-A1 study involving 266 patients with multiple myeloma who had received a median of five prior anti-myeloma regimens. The primary efficacy endpoint was overall response rate (ORR), and results showed that patients who took the drug achieved an ORR of 22.9 percent, with a median response duration of 7.8 months.

The drugmaker said safety data were evaluated in 526 patients with relapsed and/or refractory multiple myeloma who received single-agent Kyprolis, with 37 deaths recorded during the study. The most common causes of death, other than disease progression, were cardiac, end-organ failure and infection, the company said. Last month, an FDA advisory panel voted unanimously that the benefits of Kyprolis outweighed its risks, which agency reviewers had said included the potential for severe toxicities.

Onyx said enrollment has been completed for the Phase III confirmatory trial, dubbed ASPIRE, for which the company has an agreement with the FDA regarding a Special Protocol Assessment. CEO Tony Coles stated that the company is "prepared to launch, we have all the necessary plans in place."

Analysts expect the multiple myeloma market to reach $5.4 billion by 2016, with Kyprolis potentially accounting for approximately $570 million of that.

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