Rare Pediatric Disease Designation has just been granted by the FDA for an investigative therapy for lymphatic malformations (LMs). This therapy is called TARA-002 and was developed by Protara Therapeutics. This therapy has already been approved in Taiwan and Japan for the indication.

Lymphatic Malformations

Lymphatic malformations (LMs) are a rare condition that is most often congenital. The malformations in the lymphatic vessels lead to the inability of the vessels to connect in the venous system.

Rare Pediatric Disease Designation

Rare Pediatric Disease Designation is provided by the FDA for investigative therapies which have the potential to benefit children with rare and serious diseases which don’t have a strong treatment option. It includes the potential for a voucher which will provide priority review of the marketing application for a future medicine in development by the recipient company.

The designation has encouraged the medical community about the future of TARA-002. They hope that it will improve the treatment regime for this patient population. It also is a clear representation by the FDA that there is a significant need for a new option for these patients.

TARA-002 

TARA-002 is a cell-based therapy which mimics OK-432. OK-432 is the name of the therapy approved in Taiwan and Japan.

In a Phase 2 trial of OK-432, the vast majority of patients responded. To be exact, 86% of LM patients had either a substantial response or a complete response. 90% of these patients were children.

The treatment was continued long-term in this study and the positive results were sustained. 90% of the participants had no regrowth for 3 years while on the treatment.

You can read more about this therapy here.