On June 3, 2021 Protagonist Therapeutics ("Protagonist" or "the Company") (Nasdaq: PTGX), reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its lead investigational new drug candidate, rusfertide, for the treatment of patients with polycythemia vera (PV) for the reduction of erythrocytosis in those patients who do not require further treatment for thrombocytosis and/or leukocytosis (Press release, Protagonist, JUN 3, 2021, View Source [SID1234583611]). Breakthrough Therapy Designation requires that the drug candidate treat a serious or life-threatening disease or condition. It also requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation has the potential to expedite the development and regulatory review process.
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"We are thrilled to receive the Breakthrough Therapy Designation for rusfertide in PV, a serious disease where the need for different and better treatment options is clear and pressing," said Suneel Gupta, PhD, Chief Development Officer at Protagonist. "Rusfertide is a natural hormone mimetic and may stand out as the first non-cytoreductive therapeutic drug for PV. We look forward to working closely with FDA regulators to advance and complete all relevant clinical studies, both ongoing and planned, as quickly as possible."
The designation for rusfertide was supported in part by promising data from the ongoing Phase 2 clinical trial in patients with PV, presented at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in 2020. The data showed that when treated with rusfertide, a majority of patients were able to eliminate therapeutic phlebotomies, maintain a target hematocrit level of less than 45 percent, reverse iron deficiency, and experience symptom improvements. The FDA previously granted orphan drug status and Fast Track Designation to rusfertide in PV. Breakthrough Therapy Designation offers additional advantages over Fast Track Designation, including FDA actions to expedite both planned clinical trials and plans for expediting the manufacturing development strategy.
Updated data from the ongoing Phase 2 study has been selected for oral presentation at the upcoming annual meeting of the European Hematology Association (EHA) (Free EHA Whitepaper). This meeting will take place June 9 through 17 and will remain accessible until August 15, 2021.
About FDA Breakthrough Therapy Designation
Breakthrough Therapy Designation is an FDA program intended to expedite the development and regulatory review of investigational therapies that are designed to address serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation requires preliminary clinical evidence that indicates that the candidate may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. This designation provides the Company with more intensive FDA guidance on an efficient drug development program, and eligibility for other actions to expedite the FDA review, such as a rolling review of a New Drug Application (NDA), where the FDA may review sections of the NDA before the complete application is submitted. An NDA for a product candidate receiving Breakthrough Therapy Designation may also be eligible for priority review if the relevant criteria are met. Breakthrough Therapy Designation does not change the standards for approval. For more information, please visit the FDA website at www.fda.gov.