HPPI receives what would appear to be a green flag on its future NDA filing... https://finance.yahoo.com/news/hedgepath-pharmaceuticals-receives-clarity-fda-130000738.html
TAMPA, Fla., July 25, 2017 /PRNewswire/ -- HedgePath Pharmaceuticals, Inc. (HPPI), a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative therapeutics for patients with cancer, announced that the U.S. Food and Drug Administration (FDA) has provided further guidance regarding HPPI's ongoing, open-label Phase 2(b) clinical trial studying the effect of SUBA-Itraconazole (SUBA-Cap) oral capsules in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS), also known as Gorlin Syndrome.
The FDA's guidance came in the form of a written response by FDA to HPPI's Type-C meeting background package. Such a meeting is a standard element of the regulatory review process leading to a potential New Drug Application (NDA) to FDA.
Nicholas Virca, President and CEO of HPPI, stated that, "We are pleased with the FDA's guidance, since we believe it adds clarity to our regulatory and clinical road going forward for the BCCNS indication of SUBA-Cap. FDA confirmed that we may follow the more streamlined 505(b)(2) regulatory pathway, which will allow us to reference safety data from previous third-party itraconazole trials, to be supplemented by our own safety database. The acceptability of this combined safety database will then be determined by the FDA during the course of its review of the future NDA. FDA also agreed that no additional nonclinical toxicology studies appear necessary to support filing an NDA for SUBA-Itraconazole under the 505(b)(2) pathway."
Importantly, FDA also indicated that it "[w]ould accept a single study to support an NDA if results show a significant effect on a clinically meaningful endpoint. The results of the single trial must be sufficiently robust and so compelling that it would be unethical to repeat the study . . . [e]vidence of an objective reduction in tumor burden that is durable is important in order to demonstrate antitumor effects of SUBA-Itraconazole in patients with BCCNS and these data should be collected and independently reviewed."
Mr Virca further stated that, "In light of FDA's additional guidance on what might constitute a clinically significant response, we are now undertaking further detailed analyses of individual tumor responses from our ongoing trial seeking to verify the robustness of our therapy in reducing the tumour burden in BCCNS patients. We intend to present the results of this additional analysis to FDA and continue discussions with them about the utility of such results in a potential NDA submission."
Readers are cautioned that no assurances can be given that (i) the final study results will match the results previously reported on May 30, 2017 or (ii) the study, when and if completed, will achieve its primary and secondary endpoints or (iii) that the study results will be found by FDA to be sufficient for the filing of a NDA, or that one or more additional studies will not be required or (iv) if an NDA is filed, that it will be approved by FDA. Further, HPPI is not committing to providing further interim updates prior to the reporting of the final study results.