FDA Grants Orphan Drug Designation to AMXT 1501 With DFMO in Neuroblastoma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of patients with neuroblastoma, it has been announced.

The development was announced in a news release issued by Aminex Therapeutics, Incl. a clinical-stage biotechnology company.

“Receiving orphan drug designation for AMXT 1501 in combination with DFMO represents an important milestone in our mission to develop innovative therapies for children with life-threatening cancers,” said Mark Burns, chief scientific officer and president of Aminex Therapeutics, Inc., in the news release. “We are committed to working closely with regulators, investigators and patient advocacy groups, to accelerate the clinical development efforts for AMXT 1501 in combination with DFMO for the treatment of neuroblastoma and other childhood and adult tumor types in collaboration with the Beat Childhood Cancer Research Consortium.”

The FDA, according to its website, has the authority to grant orphan drug designation to a drug or biological product intended to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees and a potential seven years of market exclusivity following the drug’s approval.

“Neuroblastoma is a rare childhood cancer that unfortunately, accounts for 12% to 15% of all pediatric cancer deaths in the United States,” said Dr. Giselle Sholler in the news release. “We believe this combination has the potential to build upon the FDA approval of DFMO to further improve clinical outcomes for children with neuroblastoma and other rare childhood cancers.”

Sholler is the chief of the division of Pediatric Hematology and Oncology at Penn State Health Children’s Hospital, director of Pediatric Oncology Research and professor of Pediatrics and Neuroscience at Penn State College of Medicine and founder and chair of the Beat Childhood Cancer Research Consortium,

The Beat Childhood Cancer Research Consortium at Penn State College of Medicine, as detailed in the news release, is initiating a phase 1/2 clinical trial among pediatric patients who will be administered AMXT 1501 in combination with DFMO.

The trial, according to its listing on clinicaltrials.gov, will enroll approximately 289 patients, and has an estimated primary completion date of February 2033 followed by an estimated study completion date of February 2035.

Furthermore, Aminex Therapeutics is currently preparing to launch clinical trials to further evaluate the safety and effectiveness of AMXT 1501 in combination with metastatic melanoma and in breast cancer.

More About AMXT 1501 and DFMO

The combination treatment is an immunotherapy approach combining a polyamine uptake inhibitor, AMXT 1501, with a polyamine synthesis inhibitor, DFMO, according to the Aminex Therapeutics, Inc. website. The company stated that in multiple animal trials, the combination of AMXT 1501 and DFMO was found to support the activation of the immune system to attack and clear cancer primary tumors and to inhibit their metastases.

“AMXT 1501 [plus] DFMO negates two major immune system suppressors — polyamines and myeloid-derived suppressor cells. Further, it compromises the cancer-driving roles of both the MYC and RAS oncogenes, thought to be involved with more than sixty percent of solid tumors,” the company explains on its website.

Key Facts About Neuroblastoma

Neuroblastoma, as explained by the American Cancer Society on its website, is a rare cancer of neuroblasts, or early nerve cells, that is the most common cancer in infants and most commonly occurs in children younger than 5 years old.

The are, the American Cancer Society stated, approximately 600 to 800 new cases of neuroblastoma every year in the United States.

References

1. “Aminex Therapeutics Announces FDA Orphan Drug Designation Granted to AMXT 1501 in Combination with DFMO for the Treatment of Neuroblastoma,” news release, https://aminextx.com/wp-content/uploads/2025/10/Aminex-ODD-Press-Release-Final-10_2_25.pdf
2. “Designating an Orphan Product: Drugs and Biological Products,” FDA, https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
3. “Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas,” https://clinicaltrials.gov/study/NCT06465199
4. “Aminex Therapeutics’ immunotherapy approach combines AMXT 1501 and DFMO,” https://aminextx.com/clinical-trials/
5. “Neuroblastoma,” https://www.cancer.org/cancer/types/neuroblastoma.html

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