FDA Grants Orphan Drug Designation to Iniparib in the Treatment of Malignant Glioma Brain Tumors

Survival for patients with glioblastoma tumors is poor, with close to 75% of patients dying within 2 years of diagnosis and 95% dying within 5 years.

Officials with the FDA have granted orphan drug designation to iniparib (TriAct Therapeutics) for the treatment of patients with malignant glioma brain tumors. According to the FDA, the designation granted to iniparib is broader than the glioblastoma indication originally proposed for the therapy, with the treatment of glioblastoma falling within the scope of the orphan drug designation granted.

“With a 3-year survival rate of 16% for newly diagnosed glioblastoma patients, this designation is also an acknowledgement of the urgent need to intelligently but expeditiously develop promising new therapies for these patients. Consequently, following FDA guidance from our end-of-phase 2 meeting earlier this year, we are also filing for Fast Track designation,” said Tom White, co-founder and CEO of TriAct, in a press release.

Glioblastoma tumors are both the most aggressive form of brain cancer and the most common. Annual incidence of glioblastoma in the United States is around 3 per 100,000 population, affecting approximately 15,000 patients. Survival for patients with glioblastoma tumors is poor, with close to 75% of patients dying within 2 years of diagnosis and 95% dying within 5 years. The current standard of care is comprised of surgical resection of the tumor followed by adjuvant radiotherapy combined with temozolomide (TMZ), with subsequent maintenance TMZ.

Iniparib is a clinical stage drug candidate that targets the redox metabolism of cancer cells in order to kill them through the spiking of oxidative stress levels to trigger programmed cell death. The phase 2 study in patients with newly diagnosed glioblastoma met its primary end points both in terms of safety and survival. TriAct is currently in the process of designing a pivotal trial to support the submission of a new drug application to the FDA, and seeking drug approval in Europe.

REFERENCE

TriAct Therapeutics Announces Iniparib Granted Orphan Drug Designation by the FDA for Treatment of Malignant Glioma [news release]. TriAct Therapeutics; July 7, 2021.