FDA Grants Rare Pediatric Disease Designation to TriAct Treatment for Pediatric Diffuse High-Grade Gliomas

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to TriAct Therapeutics’ novel, proprietary TrxR1 inhibitor, iniparib, for the treatment of patients with pediatric diffuse high-grade gliomas.

“Receiving this rare pediatric disease designation, on top of the FDA’s earlier orphan drug designation, is another important milestone in our effort to bring this promising new therapy to both pediatric and adult patients suffering from malignant gliomas,” said Tom White, co-founder and CEO of TriAct. “Our initial pediatric focus is on DIPG/DMG where only 10 percent of patients survive for two years after diagnosis and there is an urgent need to develop promising new therapies such as iniparib.”

Pediatric type diffuse high-grade gliomas (pdHGG) are a group of rare, rapidly progressing tumors, affecting approximately 2,500 patients annually in the United States. Median age of the DIPG/DMG sub-group of pdHGG patients is 6 to 9 years and 3 to 10 years respectively. There are no FDA approved therapies currently and surgical resection is generally not an option due to tumor location. Standard of care is typically radiation alone. TrxR1 expression is five to 10 times greater in pdHGG tissue and CNF vs health tissue and CNF. TrxR1 over expression has been shown in published studies to desensitize tumor cells to radiation therapy.

TriAct’s lead program, iniparib, is a small molecule, brain penetrant inhibitor of TrxR1 being developed to treat an extensive list of TrxR1 over-expressing tumors, including pediatric high-grade gliomas, newly diagnosed glioblastoma and triple negative breast cancer with CNS metastases. Over expression of TrxR1 has been shown in several published studies to be prognostic for poor or worse survival in a wide range of tumors. Seven completed clinical trials in TrxR1 overexpressing tumor types met their primary efficacy endpoints, including iniparib’s phase 2 study in newly diagnosed glioblastoma. Approximately 2,900 patients have been dosed to date, with iniparib being well tolerated. Based in part on these promising results, TriAct is designing pivotal trials in TrxR1 overexpressing cancers intended to support submission of one or more NDAs in the U.S. and MAAs in Europe.

Orphan drug designation is given to experimental therapies designed to treat diseases that affect fewer than 200,000 people in the United States. Such status provides benefits that notably include a seven-year period of market exclusivity in the U.S. upon treatment approval. An additional half year US market exclusivity is added if the therapy’s initial approval is for a rare pediatric disease.

Rare pediatric disease designation is granted to promising experimental therapies for rare disorders that mainly affect children. Recipients of this designation qualify for a priority review voucher if the therapy is ultimately approved, in addition to meeting other conditions. The voucher may be used to request priority review of a future application to the FDA, or it may be transferred or sold. Most recently, Marinus Pharmaceuticals sold its voucher to Novo Nordisk for $110 million.

Photo: Tom White, co-founder and CEO of TriAct

Author: Rare Daily Staff