Ziopharm Oncology Inc (NASDAQ:ZIOP) has received a Rare Paediatric Disease Designation from the FDA for Ad-RTS-hIL-12 with veledimex in diffuse intrinsic pontine glioma (DIPG) treatment. DIPG is a lethal brain tumor in the pontine region, and it accounts for around 10% to 15% of all brain tumor cases in children.
Rare Paediatric Disease Designation to accelerated priority review of Controlled IL-12
Laurence Cooper, the company’s CEO, stated that they were pleased to receive the Rare Paediatric Disease Designation for Ad-RTShIL-12. Cooper said that currently, there isn’t any viable brain tumor treatment available, and as a result, they are working with the FDA in developing Controlled IL-12 as a new therapy for an aggressive disease that has historically been incurable.
Rare Pediatric Disease Designation is granted from life-threatening and serious diseases that affected children below 18 years and less than 200,000 people in the US. Should the Biologics Licence Application of Ziopharm for Controlled IL-12 in DIPG receive approval, then it means that the company will be eligible to get a priority review voucher from the FDA. This can be redeemed for subsequent priority marketing application review, or it can be transferred or sold to another entity for their program.
Controlled IL-12 is an investigational gene therapy program designed to limit the production of human interleukin 12, an important immune system regulator. So far, the company has treated over 175 patients with Ad-RTS-hIL-12 plus veledimex. Also, it has administered over 1,300 veledimex doses across three solid tumor types.
Ziopharm appoints new members to Scientific Advisory Board
Recently Ziopharma appointed four new members to its Scientific Advisory Board. The board led by Carl June will offer scientific and strategic counsel guiding the development of the company’s pipeline of immunotherapies for solid tumors. Appointed members include Adi Barzel, the President of the Israeli Society of Gene and Cell Therapy, Gavin Dunn, Mather Porteous Paediatrics professor at the University of Stanford, and Kole Roybal, assistant microbiology and immunology professor at the University of California.