FDA News

FDA grants orphan drug designation to SurVaxM for glioblastoma

The FDA granted orphan drug designation to SurVaxM for the treatment of patients with glioblastoma, according to the agent’s manufacturer.

SurVaxM (DRU-2017-5947, MimiVax) is an immunotherapy designed to control tumor growth and recurrence by targeting survivin, a protein that enables cancer cells to resist conventional treatments.

“There are a couple of things that distinguish our approach,” co-inventor Michael Ciesielski, PhD, assistant professor of neurosurgery at Roswell Park and chief scientific officer for MimiVax, a Roswell Park spinoff company, said in a press release. “SurVaxM is an engineered molecule capable of stimulating the immune system in several different ways to recognize and kill cancer cells. And the fact that its target, survivin, is present in many different types of cancer suggests potentially broad application against cancer.”

The FDA based the designation in part on an interim analysis from an ongoing phase 2 trial designed to evaluate SurVaxM in combination with standard treatment for patients with newly diagnosed glioblastoma.

The researchers expect to conduct late-stage clinical trials of SurVaxM.

“We are excited by the results to date and appreciative of this acknowledgement that SurVaxM holds promise,” co-inventor Robert Fenstermaker, MD, chair of neurosurgery at Roswell Park and chief medical officer at MimiVax, said in the release. “Those of us working to help patients with glioblastoma to live longer realize that the gains from existing therapies have been quite limited. We are eager to move this work forward to a larger multicenter randomized study with the momentum provided by the orphan status designation.”

SurVaxM is also being investigated to treat other malignancies. One ongoing study is designed to evaluate SurVaxM as part of combination therapy for multiple myeloma.

The FDA 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 designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

The FDA granted orphan drug designation to SurVaxM for the treatment of patients with glioblastoma, according to the agent’s manufacturer.

SurVaxM (DRU-2017-5947, MimiVax) is an immunotherapy designed to control tumor growth and recurrence by targeting survivin, a protein that enables cancer cells to resist conventional treatments.

“There are a couple of things that distinguish our approach,” co-inventor Michael Ciesielski, PhD, assistant professor of neurosurgery at Roswell Park and chief scientific officer for MimiVax, a Roswell Park spinoff company, said in a press release. “SurVaxM is an engineered molecule capable of stimulating the immune system in several different ways to recognize and kill cancer cells. And the fact that its target, survivin, is present in many different types of cancer suggests potentially broad application against cancer.”

The FDA based the designation in part on an interim analysis from an ongoing phase 2 trial designed to evaluate SurVaxM in combination with standard treatment for patients with newly diagnosed glioblastoma.

The researchers expect to conduct late-stage clinical trials of SurVaxM.

“We are excited by the results to date and appreciative of this acknowledgement that SurVaxM holds promise,” co-inventor Robert Fenstermaker, MD, chair of neurosurgery at Roswell Park and chief medical officer at MimiVax, said in the release. “Those of us working to help patients with glioblastoma to live longer realize that the gains from existing therapies have been quite limited. We are eager to move this work forward to a larger multicenter randomized study with the momentum provided by the orphan status designation.”

SurVaxM is also being investigated to treat other malignancies. One ongoing study is designed to evaluate SurVaxM as part of combination therapy for multiple myeloma.

The FDA 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 designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

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