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FDA Grants CV-01 Orphan Drug Designation and Fast Track Designation for the Delivery of Sonodynamic Therapy for Recurrent Glioblastoma

The Sonodynamic Therapy CV-01 administration has received Orphan Drug Designation and Fast Track Designation by the FDA for the treatment of recurrent glioblastoma.

Orphan Drug Designation and Accelerated Designation have been granted to CV-01 for the administration of sonodynamic therapy as a treatment strategy for patients with recurrent glioblastoma and other malignant gliomas, according to a press release from ‘AlpheusMedical.

Patients are currently being enrolled in a Phase 1 trial (NCT05362409) evaluating the administration of ultrasound with 5-ALA plus CV01 in a population with recurrent high-grade gliomas. The first human trial will assess the safety, optimal application and efficacy of the platform. The regimen is a non-invasive drug/device combination that has been designed to target cancer cells throughout the hemisphere via low-intensity diffuse ultrasound. The strategy can be used in an outpatient setting without the need for imaging.

“The diffuse nature of glioblastomas, often across the hemisphere, makes it an extremely difficult disease to treat. There are very few effective options, resulting in poor patient outcomes and universally fatal disease,” said Michael Schulder, MD, director of the Brain Tumor Center and principal investigator of the clinical trial at the Institute of Neurology and of Neurosurgery from Northwell Health. Press release. “Alpheus’ sonodynamic therapy allows for non-invasive, diffuse treatment across the hemisphere. It has the potential to change the landscape of high-grade glioma therapy and we are thrilled to be part of this important study. »

The therapeutic regimen under study consists of the oral administration of 5-aminolevulinic acid at a dose of 20 mg/kg every 4 weeks, plus a CV01 ultrasound every 4 weeks. The study has an estimated enrollment of 33 patients for phase 1. The primary endpoints are the incidence of adverse effects and determination of the maximum tolerated dose. Secondary endpoints include overall response rate by RANO criteria, duration of response, overall survival, and progression-free survival.

Inclusion criteria include a World Health Organization status of less than 2 at screening, a previously confirmed diagnosis of high-grade glioma, and radiographic evidence of recurrence or unifocal or multifocal tumors. Patients must also be 18 years of age or older and have a World Health Organization status of 2 or lower. Exclusion criteria include primary subtentorial or brainstem tumors, primary spinal cord tumors, hemispheric disease, or inability to undergo MRI or receive gadolinium-based contrast agents.

“The FDA’s expedited and orphan drug designations are significant milestones and underscore the importance of innovation in the brain cancer field,” concluded Vijay Agarwal, MD, CEO and Founder of Alpheus Medical. “Building on a very successful preclinical program, we believe that our property [sonodynamic therapy] The platform is a game-changer and has the potential to significantly advance the treatment of gliomas.


FDA Grants Alpheus Medical Orphan Drug and Fast Track Designations for Novel Sonodynamic Therapy for Brain Cancer. Press release. Alpheus Medical. July 13, 2022. Accessed July 14, 2022.