FDA Grants Rare Pediatric Disease Designation for Mateon’s DIPG Therapy

September 30, 2019

The U.S. Food and Drug Administration granted Mateon Therapeutics Rare Pediatric Disease designation for OT101 for the treatment of diffuse intrinsic pontine glioma (DIPG), the most common type of brainstem tumor in children.

Brainstem tumors comprise approximately 10-15 percent of all pediatric brain tumors. DIPG is the most common brainstem tumor and the second most common malignant brain tumor of childhood. DIPG has a low survival rate and no established or effective standard of care. The prognosis remains dismal, with a mean overall survival of 9 to 12 months from the time of diagnosis, a median survival time of approximately 10 months, and a two-year overall survival rate of less than 10 percent.

OT101, a first-in-class RNA therapeutic, is designed to counter the immunosuppressive actions of TGF-beta 2. In a completed phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult high-grade glioma patients, including adults with GBM. Further development of OT-101 may offer renewed hope for salvage therapy of pediatric DIPG patients.

“Our recent bioinformatics research has revealed that the TGF beta2 gene product, which is the molecular target for OT101, may serve as a target for immunotherapy in pediatric high-grade gliomas, especially DIPG,” said Fatih Uckun, chief medical officer of Mateon. “These in silico target validation data recently accepted for publication in a peer-reviewed medical journal extend the promising clinical data on the therapeutic activity of OT101 in adults and young adults and further demonstrate the potential of OT101 as a promising drug candidate in the treatment of pediatric DIPG, an orphan disease with a low survival rate and no established or effective standard of care.”  

The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years, and that affect fewer than 200,000 people. The designation makes the company eligible to receive a priority review voucher, which can be redeemed to reduce the review time for a new drug application to six months from ten months. The vouchers are potentially lucrative because they can be transferred and sold. In August 2019, AstraZeneca paid approximately $95 million to buy a priority review voucher from Swedish Orphan Biovitrum.

The FDA previously granted Orphan Drug designation to OT101. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing.

Photo: Fatih Uckun, chief medical officer of Mateon

Author: Rare Daily Staff

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