FDA Approves Rare Pediatric Disease Designation for IMX-110 for Rhabdomyosarcoma


IMX-110 is a tissue-specific therapeutic designed to accumulate at intended therapeutic sites at 3 to 5 times the rate of conventional therapeutics.

Officials with the FDA have approved a Rare Pediatric Disease Designation for IMX-110 for the treatment of pediatric patients with rhabdomyosarcoma, according to a press release from Immix Biopharma.1

IMX-110, a tissue-specific therapeutic designed to accumulate at intended therapeutic sites at 3 to 5 times the rate of conventional therapeutics, has already received Orphan Drug Designation for the treatment of soft tissue sarcoma. The Rare Pediatric Disease designation is intended for treatments of serious and life-threatening diseases that primarily impact children aged 18 years or younger and affect fewer than 200,000 individuals in the United States.1

“We are pleased by FDA’s acknowledgement of the urgent need for a safe and effective treatment for children with this devastating disease,” said Immix Biopharma Chief Executive Officer Ilya Rachman, MD, PhD, in the press release. “We are encouraged by our phase 1b/2a clinical data in soft tissue sarcoma.”1

Rhabdomyosarcoma is a high-grade, malignant neoplasm, and is the most common soft tissue sarcoma in pediatric and adolescent populations, but rarely occurs in adults, according to the press release. Approximately 20,000 children in the United States are currently diagnosed with rhabdomyosarcoma and the 5-year survival rate ranges from 20% to 30% for children in the high-risk group, in whom cancer spreads widely in the body.1

There are 4 main types of rhabdomyosarcoma: embryonal, alveolar, pleomorphic, and spindle cell or sclerosing. Some genetic conditions increase the risk of childhood rhabdosarcoma, including Li-Fraumeni syndrome, neurofibromatosis type 1, and Noonan syndrome. Signs and symptoms can include a lump or swelling that continues to grow or does not go away; crossed eyes or bulging of the eyes; headache; trouble urinating; or blood in the urine.2

“IMX-110 is a tissue-specific therapeutic that simultaneously attacks all 3 components of the tumor micro-environment, severing the critical lifelines between the tumor and its metabolic and structural support,” Rachman said in the press release. “We believe our SMARxT platform generating tissue-specific therapeutics represents a distinct alternative to the traditional ‘single target, single mutation’ development model.”1


1. US Food and Drug Administration Approves Immix Biopharma Rare Pediatric Disease Designation for IMX-110 as a Treatment for Life-Threatening Pediatric Cancer in Children. News release. Immix Biopharma; January 3, 2022. Accessed January 18, 2022. https://www.immixbio.com/2022/01/03/u-s-food-and-drug-administration-approves-immix-biopharma-rare-pediatric-disease-designation-for-imx-110-as-a-treatment-for-life-threatening-pediatric-cancer-in-children/

2. National Cancer Institute. Childhood Rhabdomyosarcoma Treatment – Patient Version. Updated December 22, 2020. Accessed January 18, 2022.

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