The designation allows Paris, France-based Carthera to use carboplatin chemotherapy in the treatment of malignant glioma. Carthera conducts this treatment through the use of SonoCloud, a device that emits ultrasound to temporarily increase the permeability of blood vessels in the brain, improving therapeutic molecule delivery.
After implantation in the skull, SonoCloud activates prior to the injection of a therapeutic agent. Several minutes of low-intensity ultrasound opens the blood-brain barrier (BBB) for six hours, increasing the concentration of therapeutic molecules in the brain.
Orphan drug designation applies to a drug or biological product used to prevent, diagnose or treat a rare disease or condition. In the case of FDA ODD, it can enable tax credits for qualified clinical trials, exemption from user fees and the potential for seven years of market exclusivity after approval.
Carthera is using carboplatin in the SONOBIRD pivotal trial for its SonoCloud device. The company enrolled the first patient in the trial earlier this year and aims to recruit 560 patients across 40 sites in Europe and the U.S.
“This is an important milestone for Carthera, with both U.S. and European authorities recognizing the significance of brain treatments when combined with our SonoCloud device. We believe that our technology can help harness the therapeutic efficacy of carboplatin to enhance the treatment of glioblastoma patients,” said Frédéric Sottilini, CEO of Carthera.
