Recently, ACT001, an original candidate drug for treating glioma developed by Professor Chen Yue's research group at Nankai University and Tianjin Accendatech Pharmaceutical Technology Co., Ltd. received Rare Childhood Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA), becoming the first original candidate drug from my country to obtain RPDD designation in the United States. Currently, ACT001 is undergoing clinical trials for multiple indications simultaneously in China, Australia, and the United States. The RPDD designation will further accelerate the clinical trials and market approval process for this candidate drug.

Diffuse entropy-type glioma (DIPG) is most common in children aged 5 to 10 years. Because the tumor is located in the brainstem and cannot be surgically removed, the clinical prognosis of the disease is extremely poor, with a median survival of only 9 to 12 months and a long-term survival rate of less than 1%. There are no approved drugs for the treatment of DIPG, and temozolomide, currently the best drug for the treatment of gliomas, has not shown significant efficacy.

“In the latest revised WHO classification of central nervous system tumors, the vast majority of diffuse endophytic pontine gliomas (DIPG) have been reclassified as diffuse midline gliomas (DMG) with H3K27 mutations, a new tumor type. This is defined as an invasive high-grade glioma located in the midline of the brain and is extremely malignant. The ACT001-approved rare disease designation for children is specifically for diffuse midline gliomas with H3K27 mutations,” said Chen Yue.

The primary indication for ACT001 is malignant gliomas, including DIPG. The urgent clinical need for the treatment of DMG is one of the key reasons why ACT001 was able to obtain orphan drug designation for DMG in the United States. It is worth noting that ACT001 previously received orphan drug designation in the United States and the European Union for glioblastoma (GBM) in 2017 and 2018, respectively.

In June of this year, several Chinese and foreign doctors who led the ACT001 clinical trial presented some of the clinical trial results of ACT001 at the American Society of Clinical Oncology (ASCO) and the Society for Neuro-Oncology (SNO) academic conferences on pediatric brain tumors. In particular, the report at the SNO conference attracted the attention of pediatric brain tumor doctors worldwide.

According to reports, referencing the results of clinical trials in Australia and the new capsule formulation, Tianjin Accendatech is also applying for adjustments to its clinical trial protocol for Chinese patients with diffuse engenerative pontine gliomas, increasing the dosage by approximately three times to 530-700 mg/m² BID, and applying to adopt the same capsule formulation as in Australia. The new capsule formulation contains the same amount of drug but is three times smaller, making it more convenient for patients to take. (Reporter: Bai Bo; Correspondent: Wu Junhui)