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.