FDA provides further guidance for HedgePath’s phase 2 study of Gorlin Syndrome capsules

HedgePath Pharmaceuticals said on Tuesday that the U.S. Food and Drug Administration (FDA) has provided further guidance regarding HPPI’s ongoing, open-label Phase 2 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.

According to the Hedge’s press release, the FDA’s guidance came in the form of a written response by FDA to HPPI’s Type-C meeting background package.

Nicholas Virca, President and CEO of HPPI, stated that, the FDA’s guidance is welcomed, and that he believes it adds clarity to the company’s 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,” Virca said.

The company pointed out that the 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.”

Virca further said: “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.”