Feb. 12, 2020: Deciphera Pharmaceuticals announced that the U.S. FDA has accepted for Priority Review its NDA seeking approval for ripretinib, the Company’s investigational broad-spectrum KIT and PDGFRα inhibitor, for the treatment of patients with advanced gastrointestinal stromal tumors (GIST).
The FDA granted Priority Review for the NDA, which provides for a six-month review, and allocated a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 13, 2020.
“The FDA’s acceptance of our NDA conveys us one step nearer to our goal of providing patients with the advanced GIST a potential new treatment option.”
Priority Review designation is for drugs that, if approved, would be considerable improvements in the safety or efficiency of the treatment, diagnosis, or prevention of the serious conditions when compared to the standard applications.
Under the PDUFA, a Priority Review targets a review time of six months compared to a standard review time of the ten months. in the past, FDA granted Breakthrough Therapy Designation (BTD) for the ripretinib for treatment of the patients with advanced GIST ,received prior treatment with imatinib, sunitinib, and regorafenib.
BTD is designed in order to expedite the development and review of drugs that are projected to treat a serious condition and preliminary clinical evidence indicates that the drug may reveal substantial improvement above available therapy.
The FDA is evaluating the NDA under the pilot program of the Oncology Center of Excellence for Real Time Oncology Review (RTOR).
This pilot program seeks to create a more reliable evaluation process to ensure that patients have access to safe and effective treatments as soon as possible, while preserving and improving the quality of the review.
You can find further information on RTOR at: https:/www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program.
Additionally, the Company has submitted additional marketing applicationsin Canada and Australia Australia for ripretinib in the advanced GIST. These submission are part of the FDA’s pilot program for the Project Orbis, and both received designations for priority review. The Project Orbis pilot programme, an initiative of the FDA Oncology Center of Excellence, is designed to provide a framework for the simultaneous submission and review among international partners of oncology products.You will find additional information concerning Project Orbis at: https://www.fda.gov/about-fda/oncology-center-excellence/project-orbis.
The NDA submission is supported by constructive results from the Company’s INVICTUS pivotal Phase 3 study of the ripretinib in advanced GIST. INVICTUS is a randomized (2:1), double-blind, placebo-controlled, international, multicenter study designed in order to evaluate the effectiveness and safety of ripretinib compared to placebo in 129 patients with the advanced GIST whose preceding therapies have included at least imatinib, sunitinib, and regorafenib. As per the report, the study completed its primary endpoint of improved progression-free survival compared to placebo in the patients with fourth-line and fourth-line plus GIST, as determined by the blinded independent central radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.
Ripretinib is an investigational tyrosine kinase switch control inhibitor that was designed to broadly inhibit KIT and PDGFRα mutated kinases by using the unique dual mechanism of action that controls the kinase switch pocket and activation loop. Ripretinib granted Fast Track Designation and Breakthrough Therapy Designation by the FDA for the treatment of the patients with advanced GIST, who have received previous treatment with imatinib, sunitinib, and regorafenib. The FDA also granted Priority Review for the NDA for the ripretinib, and allowed a PDUFA target action date of Aug. 13, 2020. Also, ripretinib granted Orphan Drug Designation for the treatment of GIST by the FDA and European Medicines Agency (EMA). For more information concerning the Company’s clinical trials with ripretinib, please visit www.clinicaltrials.gov.
https://investors.deciphera.com/news-releases/news-release-details/deciphera-pharmaceuticals-announces-us-food-and-drug
