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FDA Grants Orphan Drug Designation and Fast Track to CV-01 in Recurrent Glioblastoma

CV-01, Alpheus Medical’s novel sonodynamic therapy delivery platform, has received Orphan Drug and Fast Track Designation from the FDA for the treatment of patients with recurrent glioblastoma.

CV-01, Alpheus Medical’s novel sonodynamic therapy (SDT) delivery platform, has received orphan drug and fast track designations from the FDA for the treatment of patients with recurrent glioblastoma (GBM), according to a press release from Alpheus.1

SDT offers the possibility of non-invasively eradicating solid tumors in a targeted manner by sensitizing target tissues with a non-toxic sensitizing chemical agent and subsequently exposing the sensitized tissues to relatively low-intensity ultrasound.2

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

GBM is the most common primary malignant brain tumor, accounting for approximately 57% of all gliomas and 48% of all primary malignant central nervous system (CNS) tumors.3 The prognosis is poor, with a median survival of less than 2 years and a long -term survival is rare. However, molecular characteristics such as HDI-1 and HDI-2 mutations and MGMT methylation are associated with better outcomes.4

The 1-year relative overall survival (OS) was 41.4% for patients diagnosed in the United States between 2000 and 2014, improving to 44.6% from 2005 to 2014. The 5-year relative survival is 6.1% (95% CI, 6.0%-6.3%).5.6

An ongoing first-in-man phase 1 trial (NCT05362409) is evaluating delivery of CV-01 ultrasound for TDS in combination with 5-aminolevulinic acid (5-ALA) for glioma patients recurring high grade. The researchers plan to administer 5-ALA as a sonosensitizer before using CV-01 to deliver low-intensity, non-ablative ultrasound to deep brain regions to induce cancer cell apoptosis in 33

The primary endpoints are safety, tolerability and maximum tolerable duration. The estimated primary completion date is February 2023.

“The diffuse nature of GBM, 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. “The Alpheus SDT 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 FDA’s orphan and accelerated drug programs streamline the process of developing new therapies and expedite the delivery of treatments to patients with serious and rare diseases. Orphan drug status is reserved for experimental therapies for diseases or conditions affecting fewer than 200,000 people in the United States. Fast track designation facilitates communication between the FDA and the product sponsor throughout the development and review process.


  1. FDA Grants Alpheus Medical Orphan Drug and Fast Track Designations for Novel Sonodynamic Therapy for Brain Cancer. Alpheus Medical Inc. Press release. July 13, 2022. Accessed July 13, 2022.
  2. McHale AP, Callan JF, Nomikou N, Fowley C, Callan B. Sonodynamic therapy: Concept, mechanism and application to cancer therapy. Adv Exp Med Biol. 2016;880:429-50. doi:10.1007/978-3-319-22536-4_22
  3. Ostrom QT, Gittleman H, Truitt G, et al. CBTRUS Statistical Report: Primary Brain Tumors and Other Central Nervous System Tumors Diagnosed in the United States in 2011-2015. Neuro Oncol. 2018;20(suppl_4):iv1-iv86. doi:10.1093/neuonc/noy131
  4. Tan AC, Ashley DM, Lopez GY, et al. Management of glioblastoma: state of the art and future directions. CA Cancer J Clin. 2020;70(4):299-312. doi: 10.3322/caac.21613.
  5. Birzu C, French P, Caccese M, et al. Recurrent glioblastoma: from the molecular landscape to new therapeutic perspectives. Cancers (Basel). 2020;13(1):47. doi:10.3390/cancer13010047
  6. Ostrom QT, Truitt G, Gittleman H, Brat DJ, Kruchko C, Wilson R, Barnholtz-Sloan JS. Relative survival after diagnosis of a primary brain tumor or other central nervous system tumor in the National Cancer Registry Program, 2004 to 2014. Neuro-oncol practice. 2020;7(3):306-312. doi:10.1093/nop/npz059
  7. Study to evaluate 5-ALA combined with CV01 ultrasound delivery in recurrent high-grade glioma. Accessed July 13, 2022.