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Oncogenic osteomalacia is characterized by the development of a tumor that causes the bones to be weakened. This occurs when a tumor secretes a substance called fibroblast growth factor 23 (FGF23). FGF23 inhibits the ability of the kidneys to absorb phosphate. Phosphate is important for keeping bones strong and healthy. Therefore, this disease is characterized by a softening and weakening of the bones (osteomalacia). The disease also results in multiple biochemical abnormalities including high levels of phosphate in the urine (hyperphosphaturia) and low levels of phosphate in the blood (hypophosphatemia). The majority of tumors that cause oncogenic osteomalacia are small and slow-growing. These tumors most commonly occur in the skin, muscles, or bones of the extremities or in the paranasal sinuses around the head. Most of these tumors are benign, meaning they are not associated with cancer. The exact reason that the tumors associated with oncogenic osteomalacia develop is not known. The disease is diagnosed when a person experiences clinical features such as bone weakening and hyperphosphaturia and a tumor is found by imaging of the body. Treatment of the disease consists of surgical removal of the tumor. The symptoms of the disease, including the weakening of the bones, typically resolve once the tumor is removed.
Biomarker and diagnostic research for Oncogenic osteomalacia has been reported in the published literature.
No approved treatments are currently available for Oncogenic osteomalacia. An additional 1 compound holds orphan drug designation.
While no drugs are FDA-approved specifically for Oncogenic osteomalacia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for Oncogenic osteomalacia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: 1-9 in 1,000,000 (Rare).
2 clinical trials registered. Interventions under study include drug therapy and other interventions. Pipeline includes 1 NA. Research is sponsored by a mix of industry and academic institutions.
133 publications have been identified in PubMed for Oncogenic osteomalacia. Research spans Case Report / Case Series (53%), Review / Meta-Analysis (25%), and Other (5%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 70 | 53% |
Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 4:50 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Common questions about Oncogenic osteomalacia
Designated
Exclusivity End |
|---|
Designation Status |
|---|
Crysvita | burosumab-twza | Kyowa Kirin, Inc. | 2017 | 2027 | Designated (drug approved for other indication) |
Gene therapy approaches for Oncogenic osteomalacia have been reported in the published literature.
2 trials found
Research summaries |
33 |
25% |
Other research | 6 | 5% |
Clinical study results | 6 | 5% |
Laboratory research | 6 | 5% |
Disease patterns and progression | 6 | 5% |
Testing and diagnosis research | 5 | 4% |
New treatment approaches | 1 | 1% |
Amraei R (2026). [PMID: 41938633](https://pubmed.ncbi.nlm.nih.gov/41938633/). *Clin Case Rep*. [Case Report / Case Series]
Qi L (2026). [PMID: 41663581](https://pubmed.ncbi.nlm.nih.gov/41663581/). *Osteoporos Int*. [Other]
Gao B (2026). [PMID: 42221104](https://pubmed.ncbi.nlm.nih.gov/42221104/). *Front Med (Lausanne)*. [Case Report / Case Series]
Kanani AN (2026). [PMID: 41627382](https://pubmed.ncbi.nlm.nih.gov/41627382/). *Skeletal Radiol*. [Case Report / Case Series]
Brandi ML (2026). [PMID: 41912966](https://pubmed.ncbi.nlm.nih.gov/41912966/). *Arch Osteoporos*. [Epidemiology / Natural History]
Sivakumar SP (2026). [PMID: 42247568](https://pubmed.ncbi.nlm.nih.gov/42247568/). *JBJS Case Connect*. [Case Report / Case Series]
Rajasekaran S (2026). [PMID: 41961970](https://pubmed.ncbi.nlm.nih.gov/41961970/). *JBJS Case Connect*. [Case Report / Case Series]
Tosunoğlu Z (2026). [PMID: 41869937](https://pubmed.ncbi.nlm.nih.gov/41869937/). *Mol Imaging Radionucl Ther*. [Diagnostic / Biomarker]
Dodangeh S (2026). [PMID: 41657366](https://pubmed.ncbi.nlm.nih.gov/41657366/). *J Diabetes Metab Disord*. [Case Report / Case Series]
Bharati J (2026). [PMID: 42283442](https://pubmed.ncbi.nlm.nih.gov/42283442/). *Kidney360*. [Review / Meta-Analysis]
AI-curated news mentioning Oncogenic osteomalacia
Updated Aug 6, 2026
A case report highlights bilateral pathological femoral fractures in a woman with HIV, attributed to long-term use of tenofovir disoproxil fumarate leading to osteomalacia. This finding underscores the need for monitoring bone health in patients on this medication.