Protara Therapeutics Secures FDA Breakthrough Therapy Designation for TARA-002 in Pediatric Lymphatic Malformations

Protara Therapeutics, Inc., has received both Breakthrough Therapy and Fast Track designations from the U.S. Food and Drug Administration (FDA) for TARA-002. 

The investigational cell-based therapy is intended for paediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs).

In addition, TARA-002 has been selected for the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot Program, which supports expedited manufacturing development for therapies with accelerated clinical pathways.

TARA-002 is an inactivated, genetically distinct strain of Streptococcus pyogenes that retains immune-stimulating properties. 

Developed from the same master cell bank as OK-432, a therapy approved in Japan for LMs for over 30 years, TARA-002 has previously received Rare Pediatric Disease designation.

LMs are rare congenital malformations of lymphatic vessels, most commonly affecting the head and neck in young children. 

Severe cases can cause airway obstruction, bleeding, nerve or vessel impingement, recurrent infections, and functional or cosmetic issues.

The FDA designations recognise TARA-002’s potential to address significant unmet needs in this underserved patient population and aim to accelerate its clinical development and regulatory review.