Protara Therapeutics Gains FDA Breakthrough Therapy and Fast Track Status for TARA-002 in Children with Lymphatic Malformations
Tuesday, January 06, 2026
Protara Therapeutics has received both Breakthrough Therapy and Fast Track designations from the US Food and Drug Administration (FDA) for TARA-002, an investigational cell-based therapy for children with macrocystic and mixed cystic lymphatic malformations.
Alongside these designations, the FDA has selected TARA-002 for its Chemistry, Manufacturing, and Controls Development and Readiness Pilot programme. This initiative supports therapies with accelerated clinical timelines by strengthening manufacturing development and helping bring treatments to patients sooner.
Breakthrough Therapy designation is granted to medicines that show early clinical evidence of a meaningful improvement for serious conditions with limited treatment options. Fast Track status is intended to speed up the development and review of therapies that address unmet medical needs.
TARA-002 had previously received Rare Pediatric Disease designation for lymphatic malformations, reflecting the lack of effective treatment options for this condition.
The CMC Development and Readiness Pilot programme encourages early and structured engagement between companies and the FDA on manufacturing strategies. Participation allows developers to scale up production while clinical studies are still ongoing. Under this programme, manufacturing activities for TARA-002 will support both the lymphatic malformation programme and Protara’s non-muscle invasive bladder cancer development work.
TARA-002 is an inactivated, immune-stimulating strain of streptococcus pyogenes. It is derived from the same master cell bank as OK-432, a therapy approved in Japan for the treatment of lymphatic malformations and used as standard care there for more than 30 years. OK-432 was also evaluated in a large Phase II study involving over 500 patients, where it showed strong clinical benefit.
With the addition of Breakthrough Therapy and Fast Track designations, TARA-002 now holds Rare Pediatric Disease, Breakthrough Therapy and Fast Track status from the FDA for the treatment of lymphatic malformations.
Source: protaratx.com
