On February 14, 2018, the U.S. Food and Drug Administration (FDA) approved Janssen Therapeutics’ Erleada (apalutamide), a non-steroidal antiandrogen for the treatment of castration-resistant non-metastatic prostate cancer. According to the FDA, Erleada is the first oncology drug to include the endpoint of metastasis-free survival, showing a robust effect in both prolonging lifespan and preventing metastases in affected individuals. Secondary endpoints included significant improvements in time to metastasis, progression-free survival, and time to symptomatic progression. In one pivotal trial, Erleada was shown to be superior to both gonadotropin-releasing hormone analog therapy, and surgical castration, with regard to enhancing metastasis-free survival.

Erleada had previously been granted Priority Review, a designation under which the FDA seeks to take action on an application within 6 months, rather than the usual 10 months or more under standard review. Enacted under the Prescription Drug User Fee Act (PDUFA) of 1992, priority review can be requested for drugs that are expected to yield significant improvements in safety or efficacy of treatment of serious conditions, as compared to the current standard of care. In the case of Erleada, strong results from the pivotal Phase III study provided clear evidence of increased effectiveness to support expediting the review.

The Erleada approval is good news for the approximately 10-20% of prostate cancer patients suffering from the castration-resistant form of the disease. Additionally, it is noteworthy that Erleada will be the first participant in the FDA’s new Clinical Data Summary Pilot Program. This program, part of a current effort to improve transparency in drug approvals, provides for the release of curated summaries of clinical results to be posted with the Erleada entry on Drugs@FDA as well as on the pilot program landing page. While the summaries will not include raw study data, they will include clinical study report sections including information about the study protocol and statistical analysis plan for each study, as well as information on study results. The hope is that the availability of summary reports having the FDA’s imprimatur will make clinical trial results more available to prescribers, patients, advocacy groups, and other stakeholders in the drug approval process.

Written by: Dan Yarbrough, Ph.D and Jason Jardine