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Midatech Pharma brain cancer therapy gets US FDA orphan drug tag

Midatech Pharma PLC said the U.S. Food and Drug Administration granted orphan drug status to MTX110, its investigational brain cancer therapy.

The Cardiff, U.K.-based company is developing MTX110 as a potential treatment for malignant glioma, a form of cancer that forms in the brain and spinal cord.

MTX110 is also intended to treat diffuse intrinsic pontine glioma, or DIPG, a rare and fatal type of childhood brain cancer. In DIPG, the fast-growing tumor forms in the brain stem and patients have an average life expectancy of seven to nine months.

Midatech is evaluating MTX110 in an ongoing combined early and mid-stage study involving patients with DIPG.

The company is also examining the drug in other brain cancer indications, including medulloblastoma — which is most common in children and young adults — and glioblastoma multiforme, a fast-growing type of the disease that occurs in adults.