Coveted RPD designation for Ziopharm's glioma treatment

US biotech Ziopharm Oncology’s (Nasdaq: ZIOP) shares were up more than 14% at $2.83 by late morning, after it revealed announced that the US Food and Drug Administration has granted Rare Pediatric Disease designation to Ad-RTS-hIL-12 with veledimex (Controlled IL-12) for the investigational treatment of diffuse intrinsic pontine glioma (DIPG), a lethal brain tumor occurring in the pontine region of the brain.

DIPG accounts for approximately 10% to 15% of all cases of brain tumors in children. The Rare Pediatric Disease Designation program is intended to encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

“We are delighted to have received the Rare Pediatric Disease Designation for Controlled IL-12 from the FDA. This milestone for Ziopharm emphasizes the significant unmet need for children living with DIPG,” said Dr Laurence Cooper, chief executive of Ziopharm, noting: “Currently, there are no viable treatment options for this type of brain tumor. We are working with the FDA to advance Controlled IL-12 as a new gene therapy for this aggressive disease, which has historically been largely seen as incurable.”