Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation

According to a story from Biotech 365, the immuno-oncology company Mateon Therapeutics Inc. has recently announced that the company’s investigational product candidate OT101 has earned the US Food and Drug Administration’s Rare Pediatric Disease designation. OT101 is being developed as a treatment for diffuse intrinsic pontine glioma (DIPG), a rare brain tumor that mostly affects children.

About Diffuse Intrinsic Pontine Glioma

Diffuse intrinsic pontine glioma is a very rare brain tumor that is known for its poor response for treatment. It appears in the pons, the central area of the brain stem. Its locations makes the tumor inoperable via surgery. Unfortunately, the cause of this tumor remains unknown, and there are few known definitive risk factors; mutations of H3K27M are implicated in brain tumors in children. Symptoms of this cancer include vision loss, difficulty swallowing, difficulty breathing, and problems with speech. The normal treatment approach for this tumor is radiation therapy for a period of six weeks; surgery is rarely possible, and the effectiveness of chemotherapy is unclear. Like other brain tumors, this illness is difficult to treat as many drugs cannot cross the blood-brain barrier. The tumor almost always relapses after treatment, and five year survival rate is less than one percent. To learn more about diffuse intrinsic pontine glioma, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is typically reserved for therapies that are in development for rare diseases that primarily affect people under eighteen years of age. A rare disease is defined as any illness that affects less than 200,000 people in the US. Under this designation, the FDA will award priority review vouchers for a drug sponsor whose product gains approval and is designed to treat a rare pediatric disease. This voucher can subsequently be used by the company for a future approval.

About OT101

OT101 is designed to intervene in the activities of TGF-beta 2, which can suppress the immune system. The experimental drug has shown signs of efficacy in a phase 2 trial that tested the drug on high grade glioma brain tumors. The investigational drug will eventually be tested for diffuse intrinsic pontine glioma specifically as well.

 


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