FDA approves Kyprolis

Onyx Pharmaceuticals Inc. (NASDAQ:ONXX) gained $7.98 (12%) to $76.38 on Friday after FDA granted accelerated approval to an NDA for Kyprolis carfilzomib to treat patients with multiple myeloma who have received at least two prior lines of therapy that include a bortezomib and an immunomodulatory agent, and have demonstrated disease progression on or within 60 days of completion of the last therapy. The drug will be available by Aug. 1 at a price of $1,658 per vial, which the company said translates to a price of $9,950 per 28-day cycle assuming a 78 kg individual.

As part of accelerated approval, Onyx is required to complete the Phase III ASPIRE trial to confirm the selective proteasome inhibitor's clinical benefit. The trial, which has completed enrollment, is slated to finish in December 2013, with a final report due to FDA by June 2014. Onyx also agreed to use the ongoing Phase III ENDEAVOR trial to evaluate potential cardiac, pulmonary and renal toxicities as part of the agency's postmarketing requirements. In June, FDA's Oncologic Drugs Advisory Committee voted 11-0, with one abstention, that Kyprolis has a favorable benefit-risk profile based on the drug's 22.9% overall response rate in a pivotal single-arm Phase II trial (see BioCentury, June 25). ...