Protara Therapeutics Receives FDA Breakthrough Therapy and Fast Track Designations for TARA-002 in Pediatric Lymphatic Malformations
The U.S. FDA granted both Breakthrough Therapy and Fast Track designations to TARA-002, Protara Therapeutics' investigational cell-based therapy, for treating pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs) on January 5, 2026.312
TARA-002 was also selected for the FDA's Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program to accelerate development and manufacturing.312
Protara plans to meet with the FDA in the first half of 2026 to define the registration path for TARA-002 in LMs, as stated by CEO Jesse Shefferman.312
TARA-002, derived from the same master cell bank as OK-432 (standard of care in Japan for 30 years with success in a Phase 2 trial of over 500 LM patients), previously received Rare Pediatric Disease designation.324
These designations highlight the unmet need in pediatric LMs and aim to expedite access to this promising therapy.314
Sources:
1. https://www.gurufocus.com/news/4094109/protara-therapeutics-tara-gains-fda-designations-for-tara002
2. https://www.investing.com/news/company-news/fda-grants-breakthrough-therapy-designation-to-protaras-lm-treatment-93CH-4429827
3. https://www.globenewswire.com/news-release/2026/01/05/3212690/0/en/Protara-Therapeutics-Receives-Both-FDA-Breakthrough-Therapy-and-Fast-Track-Designations-for-TARA-002-in-Pediatric-Patients-with-Lymphatic-Malformations.html
4. https://drug-dev.com/protara-therapeutics-receives-both-fda-breakthrough-therapy-fast-track-designations-for-tara-002-in-pediatric-patients-with-lymphatic-malformations/