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


CV-01 delivery of sonodynamic therapy has been granted orphan drug designation and fast track designation by the FDA for the treatment of recurrent glioblastoma.

Orphan drug designation and fast track designation was granted to CV-01 delivery of sonodynamic therapy as a treatment strategy for patients with recurrent glioblastoma and other malignant gliomas, according to a press release from Alpheus Medical.

Patients are currently being enrolled in a phase 1 trial (NCT05362409) evaluating 5-ALA plus CV01 delivery of ultrasound in a population with recurrent high-grade gliomas. The first-in-human trial will evaluate the safety, optimal application, and efficacy of the platform. The regimen is a non-invasive drug/device combination that was designed to target cancer cells across the entire 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 challenging disease to treat. There are very few effective options, leading to poor patient outcomes, and a universally fatal disease,” Michael Schulder, MD, director of the Brain Tumor Center and Primary Investigator for the clinical trial at Northwell Health's Institute for Neurology and Neurosurgery, said in the press release. “Alpheus’ sonodynamic therapy enables non-invasive, diffuse treatment across the hemisphere. It has the potential to change the landscape of high-grade glioma therapy and we are excited to be part of this important study.?

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

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

“The FDA fast track and orphan drug designations are significant milestones and highlight the importance of innovation within the field of brain cancer,” Vijay Agarwal, MD, chief executive officer and founder at Alpheus Medical, concluded. “Built on a very successful pre-clinical program, we believe our proprietary [sonodynamic therapy] 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. News Release. Alpheus Medical. July 13, 2022. Accessed July 14, 2022. https://prn.to/3RtGHOJ

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