The US Food and Drug Administration (FDA) on Wednesday approved Janssen’s Erleada (apalutamide), which is the first treatment approved for patients with prostate cancer that has not spread (non-metastatic), but that continues to grow despite treatment with hormone therapy (castration-resistant).
FDA touted the first approval and the use of a novel endpoint, though Richard Lehman, senior research fellow at the Department of Primary Health Care at the University of Oxford in the UK, recently criticized the use
of a placebo in the trial.
While noting the “really impressive effect size” in the Janssen trial, Lehman said, “if apalutamide was first in a new class of anti-androgen non-steroidal drugs, it would be hailed as a breakthrough. But of course, it isn’t. The first drug in this class was flutamide (1983) and the standard since the late 1990s has been bicalutamide, which costs less than £10 per month in the UK. Apalutamide is a me-too drug and as such needs to prove its superiority over others in the class: but in this manufacturer funded study, it was compared with placebo rather than bicalutamide.
“I searched for a justification in the accompanying review article
on the management of prostate cancer but I couldn’t find any. But ethics approval was obtained in 26 countries, so I must be missing something,” he wrote.
FDA, meanwhile, said the safety and efficacy of Erleada was based on a randomized clinical trial of 1,207 patients with non-metastatic, castration-resistant prostate cancer. The median metastasis-free survival for patients taking Erleada was 40.5 months, compared to 16.2 months for patients taking a placebo, FDA said.
“This approval is the first to use the endpoint of metastasis-free survival, measuring the length of time that tumors did not spread to other parts of the body or that death occurred after starting treatment,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “In the trial supporting approval, Erleada had a robust effect on this endpoint. This demonstrates the agency’s commitment to using novel endpoints to expedite important therapies to the American public."
In addition to the approval
, Janssen will be the first participant in FDA’s recently-announced Clinical Data Summary Pilot Program
, which offers more transparency on the agency’s decision-making process. Soon after approval, certain information from the clinical summary report will post with the Erleada entry on Drugs@FDA
and on the new pilot program landing page