The US Food & Drug Administration grants this status to discoveries that target rare diseases affecting fewer than 200,000 US patients annually where there is an unmet need.
There are benefits to being singled out in this way, including accelerated approval, extended exclusivity after approval, the waiver of certain administrative fees and potential tax credits.
Midatech chief executive Craig Cook said the orphan designation for MXT110 represented an “important milestone” for the company's programme.
Currently in phase I/II, the drug is being developed to treat malignant glioma, including diffuse intrinsic pontine glioma, a fatal childhood brain cancer.
The potential treatment is a ‘solubilised’ formulation of a Novartis cancer drug called panobinostat that uses Midatech’s MidaSolve technology.
“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," CEO Cook said in a statement.