Kyprolis for multiple myeloma receives FDA approval under accelerated approval program

The safety and effectiveness of Kyprolis, which is administered directly into a patient's vein (intravenously), was evaluated in 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 (overall response rate). The overall response rate was 23 percent. The median duration of response was 7.8 months. The most common side effects observed in more than 30 percent of the study participants were fatigue, low blood cell count and blood platelet levels, shortness of breath, diarrhea, and fever. Serious side effects seen with Kyprolis included heart failure and shortness of breath. Patients should be monitored closely and treatment withheld if these serious side effects occur. 

The drug 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 required to submit additional clinical information after approval to confirm the drug's clinical benefit. 

Krypolis is marketed by Onyx Pharmaceuticals of South San Francisco, Calif. 

Source: http://www.fda.gov