FDA News

FDA grants orphan drug designation to Ad-RTS-hIL-12 for malignant glioma

The FDA today granted orphan drug designation to Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex, or Ad-RTS-hIL-12,  for the treatment of malignant glioma, according to a press release from the drug’s manufacturer.

Ad-RTS-hIL-12 (Ziopharm) — a novel gene therapy candidate for the controlled expression of interleukin 12, a protein that stimulates an anti-cancer T cell immune response — is currently being evaluated in a phase 1 trial of patients with high-grade gliomas.

The study’s primary objective is to evaluate the safety and efficacy of a single intra-tumoral Ad-RTS-hIL-12 injection plus escalating dose of oral veledimex. The study’s secondary objectives include determining the maximum tolerated dose of veledimex, the immune responses caused by Ad-RTS-hIL-12 plus veledimex, tumor objective response rate, PFS and OS, according to the press release.

Up to 72 subjects are expected to enroll in the study, which will be conducted in 12 treatment centers.

"Malignant glioma is an aggressive cancer with few treatment options," Laurence J. N. Cooper, MD, PhD, CEO of Ziopharm, said in a press release. "This designation supports our effort to advance Ad-RTS-IL-12, a novel immuno-oncology therapy which has demonstrated promising preclinical efficacy in brain cancer, as a potential treatment for this indication. Enrollment in our phase 1 study is progressing well, and we look forward to early results from this clinical trial later in the year."

The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

The FDA today granted orphan drug designation to Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex, or Ad-RTS-hIL-12,  for the treatment of malignant glioma, according to a press release from the drug’s manufacturer.

Ad-RTS-hIL-12 (Ziopharm) — a novel gene therapy candidate for the controlled expression of interleukin 12, a protein that stimulates an anti-cancer T cell immune response — is currently being evaluated in a phase 1 trial of patients with high-grade gliomas.

The study’s primary objective is to evaluate the safety and efficacy of a single intra-tumoral Ad-RTS-hIL-12 injection plus escalating dose of oral veledimex. The study’s secondary objectives include determining the maximum tolerated dose of veledimex, the immune responses caused by Ad-RTS-hIL-12 plus veledimex, tumor objective response rate, PFS and OS, according to the press release.

Up to 72 subjects are expected to enroll in the study, which will be conducted in 12 treatment centers.

"Malignant glioma is an aggressive cancer with few treatment options," Laurence J. N. Cooper, MD, PhD, CEO of Ziopharm, said in a press release. "This designation supports our effort to advance Ad-RTS-IL-12, a novel immuno-oncology therapy which has demonstrated promising preclinical efficacy in brain cancer, as a potential treatment for this indication. Enrollment in our phase 1 study is progressing well, and we look forward to early results from this clinical trial later in the year."

The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.