Aminex Therapeutics receives second FDA orphan drug designation for AMXT 1501 targeting malignant glioma including DIPG
Friday, March 13, 2026
Aminex Therapeutics, Inc., a clinical-stage biotechnology company developing metabolic-targeted cancer therapies, has received Orphan Drug Designation from the U.S. Food and Drug Administration for AMXT 1501 in combination with Difluoromethylornithine (DFMO). The designation covers the treatment of malignant glioma, including Diffuse Intrinsic Pontine Glioma (DIPG).
This is the company’s second orphan designation, following a similar status granted in October 2025 for Neuroblastoma.
DIPG is a highly aggressive brainstem tumour that mainly affects children aged between five and ten. The disease has a very poor prognosis, with median survival typically less than one year after diagnosis, and there is currently no curative treatment.
A national Phase 1/2 clinical trial is under way to evaluate AMXT 1501 combined with DFMO in paediatric patients with DIPG, neuroblastoma, sarcomas and other high-risk childhood cancers. The study is being conducted by the Beat Childhood Cancer Research Consortium at the Penn State College of Medicine in collaboration with Aminex. The trial is expected to involve around 50 clinics across the United States.
AMXT 1501 is also being assessed in adults with solid tumours, including breast cancer and metastatic melanoma, in an ongoing Phase 1b/2 multicentre clinical study.
The orphan drug designation provides development incentives from the FDA, including tax credits for clinical research, waiver of certain regulatory fees and up to seven years of market exclusivity in the United States if the therapy receives approval.
Source: prnewswire.com
