- In a first-of-its-kind approval on Wednesday, the Food and Drug Administration approved a treatment based on a new endpoint – metastasis-free survival. Johnson & Johnson's apalutamide is now indicated for non-metastatic castration-resistant prostate cancer.
- The drug, approved ahead of its April user fee action date, will be sold under the trade name Erleada. It works by blocking certain androgens, or hormones, that can promote tumor growth.
- The approval comes less than two weeks after J&J announced positive topline results from a Phase 3 study that showed Erleada delayed the development of metastases by more than two years.
Typically cancer drugs are approved based on their ability to extend survival of patients or halt progression of disease, but the approval of Erleada was based on the drug preventing the cancer from spreading to other parts of the body.
"The FDA evaluates a variety of methods that measure a drug’s effect, called endpoints, in the approval of oncology drugs," said Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the 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."
The approval is just the latest evidence showing how the agency is shifting under Commissioner Scott Gottlieb. The FDA noted in a statement that Erleada was the first participant in the agency's recently enacted Clinical Data Summary Pilot Program. The new effort is part of the FDA's move to increase transparency and increase the dialogue with drug manufacturers.
Erleada's approval is a huge win for J&J. It is the first drug to be approved in the non-metastatic castration-resistant setting. J&J has been neck and neck with Pfizer's Xtandi (enzalutamide) in a race to grab the indication. Both companies announced positive results for their respective drugs in the setting earlier this month.
Not only will Erleada beat out Xtandi, but the approval comes at a time when J&J's blockbuster prostate cancer drug Zytiga (abiraterone) is facing challenges to its patent library. A recent patent decision could allow generic competition of the drug to enter the market earlier. Zytiga brought in more than $2.5 billion last year, leading the company's oncology portfolio.
Erleada would occur earlier in the treatment paradigm, allowing patients to take it first and then take Zytiga once their diseases progress — handing J&J a way of further dominating the market.