Deciphera Pharmaceuticals has filed for FDA approval of ripretinib in patients with advanced gastrointestinal stromal tumors (GISTs). The filing moves Deciphera closer to establishing ripretinib as an option for patients failed by drugs from Bayer, Novartis and Pfizer.
Ripretinib came through a phase 3 trial in fourth-line GIST patients earlier this year, generating data to show the broad-spectrum KIT and PDGFRα inhibitor extends progression-free survival (PFS) in a group with limited options. Median PFS in the treatment group came in at 6.3 months, as compared to one month in the placebo cohort.
Deciphera has corralled the data into a filing for FDA approval. The filing covers the use of ripretinib in patients previously treated with Bayer’s Stivarga, Novartis’ Gleevec and Pfizer’s Sutent.
The FDA will evaluate the filing under its Oncology Center of Excellence Real-Time Oncology Review (RTOR), a pilot project intended to help the agency start going over data sooner. The project enables companies to share top-line data with the FDA and, if the agency accepts them into RTOR, send presubmission data from as little as two weeks after database lock.
Officials at the FDA evaluate the presubmission data for “sufficiency and integrity” before receiving the filing for approval. The FDA provides early feedback “regarding the most effective way to analyze data to properly address key regulatory questions” and, if the program works as intended, is well positioned to efficiently review the proper filing for approval when it arrives.
Deciphera has been tight-lipped as it has gone through this process, going as far on its third-quarter results conference call last month to refuse to say whether it would make a rolling or standard filing. There are signs the process has gone well for Deciphera, though. Last month, Deciphera said it was aiming to file for FDA approval in the first quarter, a target it ultimately beat by two weeks.
Ripretinib could continue to advance swiftly from here. The FDA has granted breakthrough status to ripretinib and, having preassessed the data under its RTOR program, is potentially well placed to quickly review the complete package.
Deciphera has already started to build out the team it hopes will turn ripretinib into a commercial success, bringing people with experience launching oral oncolytics in the U.S. on board as it works to establish a group that could ultimately feature 60 sales representatives.