FDA Grants Rare Pediatric Disease Designation to Ziopharm’s Gene Therapy for DIPG

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to Ziopharm Oncology’s experimental gene therapy for the treatment of diffuse intrinsic pontine glioma, a rare and lethal brain tumor occurring in the pontine region of the brain.

Glioma in the pontine region of the brain, or DIPG, accounts for approximately 10 to 15 percent of all cases of pediatric brain tumors, with about 150-300 new diagnoses per year in the United States. Median survival ranges from 8-11 months and currently there are no curative options.

Ziopharm’s Controlled IL-12 (Ad-RTS-hIL-12 plus veledimex) platform is an experimental gene therapy designed to induce and control the production of human interleukin 12 (hIL-12), a master-regulator of the immune system. The company has treated more than 175 patients, including more than 125 patients with rGBM with Controlled IL-12, and administered more than 1,300 doses of veledimex across three types of solid tumors, building a significant safety profile, mechanistic dataset and evidence of anti-tumor effects.

Controlled IL-12 is being studied in a phase 1/2 trial designed to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 given with up to 14 days of oral veledimex in children with gliomas. Up to 12 patients with DIPG may be enrolled in phase 1 of the study, which is being conducted at leading pediatric cancer centers across the United States, including Lurie Children’s Hospital in Chicago, Dana-Farber Cancer Institute in Boston and University of California in San Francisco.

“This milestone for Ziopharm emphasizes the significant unmet need for children living with DIPG,” said Laurence Cooper, CEO of Ziopharm. “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.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease primarily affecting individuals aged 18 years or younger that impacts fewer than 200,000 people in the United States. The program is intended to facilitate development of new drugs and biologics for the prevention and treatment of these conditions.

If Ziopharm’s Biologics License Application (BLA) for Controlled IL-12 in DIPG is approved, the company may be eligible to receive a priority review voucher from the FDA, which can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: Laurence Cooper, CEO of Ziopharm

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.