R&D biotechnology company, Midatech Pharma (MTPH) , announced on Thursday that the U.S. Food and Drug Administration had granted the company orphan drug designation for its drug, MTX110.
"Receiving orphan drug designation for MTX110 is another important milestone to emerge from our innovative MTX110 programme, based on our MidaSolve technology,” said CEO, Craig Cook.
Orphan designation is granted by the FDA in support of drugs targeting rare diseases with high unmet needs.
MTX110 has been developed for the treatment of patients with Malignant Glioma, a type of tumour occurring in the brain and spinal cord -- this includes Diffuse Intrinsic Pontine Glioma ("DIPG"), a rare and fatal form of childhood brain cancer.
The drug is a novel solubilised formulation of the HDAC-inhibitor panobinostat drug that is currently being evaluated in an ongoing combined Phase 1 and Phase II programme in patients with DIPG.
Shares in Midatech Pharma jumped over 9% following this morning's news.
DIPG is a highly aggressive and inoperable tumour located in the brain stem that is universally fatal with an average life expectancy of 7 to 9 months.
Mr Cook acknowledged that promising developments were being made to advance the drug, commenting:
“We are pleased with the progress we are making in the clinic at present and hope to ultimately demonstrate that MTX110 can serve as a safe and effective treatment option for these patients. We look forward to providing further updates as the development programme progresses."
The company explained that they are also evaluating MXT110 to treat other brain cancers such as Medulloblastoma in children, as well as Glioblastoma Multiforme, a fast-growing form of brain cancer in adults.
Follow News & Updates from Midatech Pharma here: