Onyx Pharmaceuticals Inc. (ONXX) recently announced that it has completed enrollment in the late-stage ASPIRE study that is being conducted with carfilzomib. The study is evaluating carfilzomib in combination with Celgene Corp.‘s (CELG) Revlimid (lenalidomide) and low dose dexamethasone in patients with relapsed multiple myeloma (MM).
Onyx Pharma is conducting the trial under the US Food and Drug Administration’s (FDA) special protocol assessment (SPA). The company has also received scientific advice from the European Medicines Agency (EMA) on the design and planned analysis of the study.
Carfilzomib is currently under regulatory review in the US for the treatment of patients with relapsed and refractory MM. The regulatory body has granted a standard review designation to the candidate and set a target date of July 27, 2012.
Onyx Pharma was seeking to get priority review status for carfilzomib’s application. However, the FDA issued a letter in December last year, stating that the new drug application (NDA) and the study supporting the application are insufficient to review it on a priority basis. The regulatory body also said that since the NDA is based on a single-arm study, it is concerned whether the benefit and risk of carfilzomib are appropriately balanced.
It was also mentioned in the letter that the FDA’s advisory committee prefers late-stage study results for granting accelerated approval designation to a candidate’s NDA, whereas, carfilzomib’s NDA was based on mid-stage trial (003-A1) results.
Onyx Pharma is conducting another phase III study (FOCUS) to support the European filing of carfilzomib as a treatment for relapsed and refractory myeloma. Top-line data from the study is expected in the first half of 2012.
We note that the company is banking heavily on the approval of carfilzomib as currently it just has one marketed product, Nexavar (cancer), in its portfolio.
Zacks Investment Research