Onyx Pharmaceuticals Inc. (ONXX) recently announced that it has completed the submission of the rolling new drug application (NDA) to the US Food and Drug Administration (FDA) for its pipeline candidate, carfilzomib. The company is seeking approval of carfilzomib as a treatment for patients with relapsed and refractory multiple myeloma. Additionally, Onyx Pharma requested the regulatory body to review the application on a priority basis.
We note that Onyx Pharma had initiated the submission of the rolling NDA in January 2011, after the FDA granted a fast track designation to carfilzomib. The company’s NDA submission is based on positive results on the candidate from a mid-stage trial (003-A1).
The trial demonstrated that patients experienced an overall response rate of 24.1%, while the clinical benefit rate was higher at 34.2%. The duration of response was 8.3 months, while the median overall survival was 15.5 months. The patients, who enrolled in the study, had already received a median of five prior lines of therapy and their disease was refractory to their last therapeutic regimen.
We note that the FDA granted orphan drug designation to carfilzomib for the treatment of multiple myeloma in 2008. Following the drug’s approval, this status will provide Onyx Pharma with seven years of US marketing exclusivity.
Onyx Pharma is currently conducting a randomized international phase III trial (ASPIRE or 009) which will evaluate the combination of Celgene Corp.’s (CELG) Revlimid and low dose dexamethasone with or without carfilzomib in relapsed multiple myeloma patients.
The study, which is being conducted under the FDA’s special protocol assessment (SPA) program, is expected to complete enrollment in the first half of 2012. Data from the study is expected to be released in the first half of 2013.
Additionally, the company is conducting a 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.
Onyx Pharma has an agreement with a Japanese company, Ono Pharmaceutical Co. Ltd., for the development and commercialization of carfilzomib and ONX 0912, in Japan. ONX 0912, the follow-on version of carfilzomib, is currently in phase I testing. Onyx Pharma plans to initiate a mid-stage trial with ONX 0912 in the fourth quarter of 2011.
We believe that the approval of carfilzomib would be a major positive for Onyx Pharma, which currently has just one marketed product, Nexavar (cancer), in its portfolio.
