On January 5, 2026 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy and Fast Track designations for TARA-002, the Company’s investigational cell-based therapy, for the treatment of pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs). In addition, the FDA has selected TARA-002 to participate in the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program, which aims to support CMC development of products with expedited clinical development timeframes and provide patients with earlier access.
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"Receiving these important FDA designations and invitation to participate in the CDRP program highlights the significant unmet need among pediatric patients with LMs and underscores our belief that TARA-002 could serve as a meaningful treatment option for this underserved patient population," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to continuing to work with the Agency to bring this promising therapy to patients as expeditiously as possible and expect to meet with the FDA to define the path to registration for TARA-002 in LMs in the first half of this year."
The FDA’s Breakthrough Therapy designation is a process designed to expedite the development and regulatory review of drugs or biologics that are intended to treat serious conditions where preliminary clinical evidence indicates that the drug or biologic may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy. The FDA’s Fast Track program is intended to facilitate the development and expedite the review of new drugs and biologics designed to treat serious conditions with unmet medical needs.
TARA-002 was previously granted Rare Pediatric Disease designation for the treatment of LMs.
The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. The initiative is designed to promote earlier and more structured engagement between sponsors and the FDA on CMC development strategies, and since its inception, has led to increased collaboration with the FDA so that sponsors can confidently scale up manufacturing capacity while clinical development is ongoing. TARA-002 has been accepted into this program, which will involve manufacturing for both the company’s LMs and non-muscle invasive bladder cancer (NMIBC) programs.
About TARA-002 in LMs
TARA-002 is an investigational, genetically distinct strain of streptococcus pyogenes that is inactivated while retaining its immune-stimulating properties. It was developed from the same master cell bank as OK-432, which was originally granted marketing approval by the Japanese Ministry of Health for the treatment of LMs and has been the standard of care in Japan for 30 years. In addition, OK-432 was studied in a large Phase 2 trial in LMs in over 500 patients with significant clinical success. TARA-002 has been granted Rare Pediatric Disease, Breakthrough Therapy and Fast Track designations by the U.S. Food and Drug Administration for the treatment of LMs.
(Press release, Protara Therapeutics, JAN 5, 2026, View Source [SID1234661720])