The clock is ticking! Just three weeks left for an FDA advisory panel to review Onyx Pharmaceuticals Inc.'s (ONXX) investigational therapy, Kyprolis, for the treatment of patients with relapsed and refractory multiple myeloma.
Kyprolis, whose chemical name is Carfilzomib, is a next-generation proteasome inhibitor, belonging to the same class as Takeda Pharmaceutical Co.'s multiple myeloma treatment Velcade.
Onyx submitted the New Drug Application for Kyprolis in relapsed and refractory multiple myeloma last September based on a single-arm phase 2b trial under the accelerated approval process. This study, dubbed 003-A1, evaluated 266 heavily-pretreated patients with relapsed and refractory multiple myeloma who had received at least two prior therapies, including Velcade and either thalidomide or lenalidomide (Revlimid).
According to the 003-A1 study results, the clinical benefit rate, which refers to the percentage of patients who achieved complete response, partial response and stable disease, with Kyprolis was 37 percent with a duration of response of 8.3 months. Duration of response refers to length of time between treatments where a patient's tumor shrinks, disappears or remains stable. The median overall survival for all patients in the study, regardless of response to Kyprolis, was 15.6 months.
The Kyprolis NDA is scheduled to be reviewed by the Oncology Drug Advisory Committee on June 20. The final decision of the regulatory agency is set for July 27.
Kyprolis is also under two phase III clinical trials, which will help Onyx pursue full approval of the drug for patients with relapsed multiple myeloma, in the U.S. and Europe.
The first is a phase III international randomized trial, known as the ASPIRE trial, evaluating the safety and efficacy of Kyprolis in combination with lenalidomide and low-dose dexamethasone as a potential treatment for patients with relapsed multiple myeloma. This study completed the targeted enrollment of 780 patients in February of this year, and interim results are expected in the first half of 2013.
The second study, known as the FOCUS trial, is a phase III study of single-agent Kyprolis in the relapsed and refractory setting and is designed to support a regulatory filing in Europe. Onyx expects to complete enrollment in the FOCUS study in the first quarter of 2013.
The company also intends to initiate a global phase III study, dubbed ENDEAVOR, in relapsed multiple myeloma, comparing Kyprolis against sub-Q or IV Velcade in combination with low-dose dexamethasone by the middle of this year.
Multiple myeloma is a type of blood cancer, and an estimated 200,000 people worldwide are living with this disease. In the U.S, about 50,000 people are affected by multiple myeloma, and 20,000 new cases are diagnosed each year. Onyx sees over $2 billion global sales opportunity in multiple myeloma.
Will Kyprolis clear the penultimate regulatory hurdle of winning the FDA panel backing? Stay tuned...
Onyx shares have thus far hit a 52-week low of $27.17 and a 52-week high of $47.80. The stock closed Tuesday's trading at $46.73.
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