
Jul 17 2025
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FDA Grants Rare Pediatric Disease Designation to 177Lu-omburtamab-DTPA for Medulloblastoma
Y-mAbs Therapeutics, Inc., a commercial-stage biopharmaceutical company concentrated on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer; today announced that the US Food and Drug Administration (FDA) had granted Rare Pediatric Disease Designation for the Company’s lutetium labeled omburtamab antibody program for the treatment of medulloblastoma.
Source: Y-mAbs Therapeutics, Inc.
Medulloblastoma is the most ordinary malignant brain tumor in children, and it is an embryonal lesion caused by progenitor cell populations present during early brain development. It is responsible for about 20% of all juvenile brain cancers and 63% of intracranial embryonal tumors. Hereditary cancer predisposition syndromes are the most critical risk factors for its development. WNT MB can be caused by germline mutations in WNT signaling pathway genes, such as APC mutations in Turcot syndrome. WNT subgroup MB makes for about ten percent of all its cases. It is the most frequent malignant brain tumor in children aged four to fourteen years old and the second most prevalent in children aged five to fourteen years old. More than 70% of all pediatric MB are identified in children under the age of ten, with the median age of diagnosis being seven.
- The incidence of cases of Medulloblastoma ranges from 1.49 to 3.72 per million population in the USA per year.
However, the current Medulloblastoma treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (AloCELYVIR, Pomalidomide, LDE225, TB-403, ZSP1602, 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 Medulloblastoma treatment. The key companies in the advanced development stage are Guangdong Zhongsheng Pharmaceutical Co., Ltd., Novartis Pharmaceuticals, Oncurious NV, Apices Soluciones S.L., etc.
Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook report on Medulloblastoma 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.
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