BETHESDA, MD 20 July 2012—
FDA and Onyx Pharmaceuticals today announced the approval of carfilzomib in patients with multiple myeloma.
The drug will be marketed under the brand name Kyprolis. It is indicated for the treatment of multiple myeloma in patients previously treated with at least two other therapies, including bortezomib and an immunomodulatory agent, whose disease progressed within 60 days after the last treatment.
About 10,000–15,000 U.S. patients may qualify for treatment with carfilzomib, according to Onyx.
Helen Torley, the company's chief commercial officer, said during a conference call today that carfilzomib will be available through a specialty pharmacy network by August 1.
Labeling (PDF) for carfilzomib describes the drug as a tetrapeptide epoxyketone proteasome inhibitor that inhibits proliferation and increases apoptosis in tumor cells.
In the pivotal 266-patient clinical trial of carfilzomib, tumor regression occurred in 23% of patients who received up to 12 cycles of treatment with the drug.
It is not known whether treatment with carfilzomib prolongs patients' lives or increases progression-free survival.
U.S. sales of Carfilzomib are authorized under FDA's accelerated approval process, which requires the company to provide additional clinical data about the drug. Clinical studies of carfilzomib are now under way to further assess the drug's efficacy as well as safety concerns, including possible toxic effects on the heart and other organs.
In clinical trials, at least 30% of carfilzomib recipients reported fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea, or pyrexia.
Patients undergoing treatment with carfilzomib should be monitored for heart or lung complications, tumor lysis syndrome, and thrombocytopenia, according to the drug's labeling. Liver enzymes should also be monitored and therapy suspended if liver toxicity or liver failure are suspected.
Each 28-day treatment cycle of carfilzomib consists of six infusions interspersed with nontreatment intervals as described in the drug's labeling.
The recommended dosage of carfilzomib during the first treatment cycle is 20 mg/m2 of body surface area infused intravenously over 2–10 minutes. The dose should be increased to 27 mg/m2 for subsequent treatment cycles if the patient tolerates the drug.
Treatment cycles should continue until unacceptable toxicity occurs or the tumor fails to respond. A large table in the drug's labeling provides instructions for modifying dosages in response to evidence of toxicity.
During the first treatment cycle, the patient should be premedicated with dexamethasone before each infusion to reduce the risk of infusion reactions.
Carfilzomib will be supplied in 60-mg single-use vials. The drug should be refrigerated until use, then prepared for administration as described in the labeling.