Midatech Pharma PLC (AIM: MTPH.L; Nasdaq: MTP), an R&D biotechnology company focused on delivering innovative oncology and rare disease products to patients, is pleased to announce that the U.S. Food and Drug Administration (“FDA”) has granted the Company orphan drug designation for MTX110 for the treatment of patients with Malignant Glioma, including Diffuse Intrinsic Pontine Glioma (“DIPG”), a rare and fatal form of childhood brain cancer. MTX110 is a novel solubilised formulation of the HDAC-inhibitor panobinostat that is currently being evaluated in an ongoing combined Phase 1 (safety) and Phase II (efficacy) programme in patients with DIPG.
MTX110 is designed for the treatment of childhood brain cancers such as DIPG, an ultra-rare highly aggressive and inoperable tumour located in the brain stem, that is universally fatal with an average life expectancy of 7 to 9 months. Midatech is also evaluating MXT110 for the treatment of other childhood brain cancers such as Medulloblastoma, and also Glioblastoma Multiforme (“GBM”) which is a fast-growing form of brain cancer in adults.
Commenting Craig Cook, CEO of Midatech said: “Receiving orphan drug designation for MTX110 is another important milestone to emerge from our innovative MTX110 programme, based on our 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.”
Orphan designation by the FDA is granted to support the development of drugs that target rare diseases with high unmet needs, affecting 200,000 or fewer U.S. patients annually, and that are expected to provide significant therapeutic advantage over existing treatments. Orphan designation qualifies a company for benefits that apply across all stages of drug development, including an accelerated approval process, seven years of market exclusivity upon regulatory approval, if received, tax credits for qualified U.S. clinical trials, eligibility for orphan drug grants, and exemption from certain administrative fees.