FDA grants rare pediatric disease designation to IMX-110 for Rhabdomyosarcoma (RMS) treatment

Jul 18 2025

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FDA grants rare pediatric disease designation to IMX-110 for Rhabdomyosarcoma (RMS) treatment

Immix Biopharma, Inc., a biopharmaceutical company that pioneered Tissue-Specific Therapeutics (TSTx)TM for oncology and immuno-dysregulated diseases, announced today that the U.S. Food and Drug Administration (FDA) had granted IMX-110 rare pediatric disease designation for the treatment of Rhabdomyosarcoma (RMS), a life-threatening form of pediatric cancer in children. An investigational product called IMX-110 is currently being tested in Phase 1b/2a clinical trial.

Source: Immix Biopharma, Inc.

Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children. RMS can occur anywhere in the body, including the head and neck, genitourinary organs, extremities, and abdomen. Although still debated, evidence suggests that RMS arises from the mesenchymal cell lineage, which is typically fated to become skeletal muscle tissue. RMS is traditionally classified into two major histological subtypes, embryonal RMS (ERMS) and alveolar RMS (ARMS), of which 60–70% of cases are ERMS and 20–30% are ARMS. 80% of ARMS cases are defined by a chromosomal translocation between PAX3 or PAX7 and FOXO1 genes; these translocations result in fusion genes that primarily drive oncogenic activity. The other 20% of ARMS cases are similar to ERMS in terms of clinical outcomes and the pattern of somatic mutations. Despite the clinical significance of RMS, the underlying etiologies remain the environmental and genetic factors individually contribute to this malignancy.

  • RMS accounts for roughly half of all pediatric soft-tissue sarcoma diagnoses, with a rate of 4.71 per million children and adolescents under the age of 20 in the United States.

However, the current Rhabdomyosarcoma (RMS) treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (afatinib, Eribulin mesilate, LDE225, IMC-A12, nab-paclitaxel, etc.) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Rhabdomyosarcoma (RMS) treatment. The key companies in the advanced development stage are Boehringer Ingelheim, Oncurious NV, Eisai Inc., Novartis Pharmaceuticals, etc.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market insight report on Rhabdomyosarcoma (RMS) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.

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