PDGFRA across 5 cancers
PDGFRA is on the target list of 5 of the briefings. A drug aimed at it carries an FDA label naming the disease in 3, and is approved for some indication in 5. In the reference cohorts of the 5 with a mutation landscape it is altered in at least 5% of patients in 3.
| Disease | Label status | Furthest stage (Open Targets) | Distinct drugs | Cell-therapy trials | Altered in the reference cohort |
|---|---|---|---|---|---|
| Gastrointestinal stromal tumour | approved here Ripretinib | approved | 30 | 0 via PDGFRA | 11.09% mut · 1.92% amp n=469 |
| Small intestine cancer | approved here Ripretinib | approved | 30 | — | 5.26% mut n=152 |
| Soft tissue sarcoma | approved here Pazopanib | approved | 30 | 0 via PDGFR-alpha | 0.39% mut · 3.16% amp n=255 |
| Glioblastoma | approved elsewhere | approved | 30 | — | 4.1% mut · 13.04% amp n=390 |
| Lower-grade glioma | approved elsewhere | approved | 30 | 0 via PDGFR | 1.56% mut · 3.72% amp n=514 |
"Approved here" means a drug Open Targets records as aimed at PDGFRA carries an FDA label that names that disease, per the briefing's openFDA stage. "Approved elsewhere" means the drug is approved for some other indication. Stage is the furthest Open Targets clinical stage among drugs aimed at the gene, in any indication; the database is reported as it is, and it lags some approvals. Cell-therapy trials are counted on the antigen the briefing maps the gene to, from ClinicalTrials.gov. The last column is the disease's mutation landscape: non-silent mutation, high-level amplification and deep deletion as percentages of the reference cohort's patients, each over its own denominator; "not on panel" means the reference cohort could not have called the gene.