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Protara Therapeutics Receives Rare Pediatric Disease Designation for TARA-002 for the Treatment of Lymphatic Malformations
NEW YORK, July 28, 2020 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical stage company developing treatments for rare and specialty
About this update from Protara Therapeutics, Inc.
[{"type":"text","content":"NEW YORK, July 28, 2020 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical stage company developing treatments for rare and specialty diseases with significant unmet needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation for TARA-002 for the treatment of Lymphatic Malformations (LMs). TARA-002 is an investigational cell-based therapy based on the broad immunopotentiator OK-432, which is approved in Japan and Taiwan for the treatment of LMs. LMs are rare, typically congenital, malformations of the lymphatic vasculature.\n “Receipt of Rare Pediatric Disease designation from the FDA for TARA-002 in LMs further underscores the significant unmet medical need for the children that are affected by this rare and serious disease,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We are encouraged about the potential of TARA-002 to enhance the treatment paradigm of LMs for this underserved population, and we look forward to working with the agency to determine next steps for the program in the coming months.” About FDA Rare Pediatric Disease Designation The FDA grants Rare Pediatric Disease designation for serious diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the United States. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred. About TARA-002 for Lymphatic Malformations TARA-002, Protara’s lead product candidate, is an investigational cell-based therapy based on the broad immunopotentiator OK-432. OK-432 is approved in Japan and Taiwan for lymphatic malformations (LMs), which are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. In a randomized, Phase 2 clinical trial of OK-432 in LMs, 86% of patients treated with OK-432 (>90% pediatric) experienced a complete or substantial response. Long-term control of LMs was favorable, with more than 90% of patients ...