Focused Ultrasound Combination Therapy for DIPG Earns FDA’s Fast Track Designation


Key Points

  • The US Food and Drug Administration has granted Fast Track designation to the combination of SonALAsense’s proprietary drug and Insightec’s focused ultrasound device for the treatment of pediatric brain tumors.  
  • The Fast Track program facilitates the development and expedited review of drugs to fill an unmet medical need. 
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The US Food and Drug Administration (FDA) has granted Fast Track Designation for the development of SonALAsense’s proprietary aminolevulinic acid HCl (ALA) formulation, called SONALA-001, in combination with Insightec’s Exablate Neuro focused ultrasound device for the treatment of patients with diffuse intrinsic pontine glioma (DIPG).  This combination approach to cancer treatment is called sonodynamic therapy (SDT).

The FDA’s Fast Track is a process designed to facilitate the development, and expedited review, of drugs to treat serious conditions and fill an unmet medical need. The goal is to make new therapies available to patients as quickly as possible. The program allows companies to meet frequently with the FDA to review the drug’s development plan, discuss clinical trial design, and more. The designation could also make the drug eligible for priority review and accelerated approval.  

This Fast Track designation is specifically for the combination treatment of DIPG, a highly aggressive and devastating brain tumor that affects young children, typically aged 5–9. Only 10 percent of children survive past the two-year mark after diagnosis because current standard of care therapies are highly ineffective. 

SonALAsense is currently sponsoring a clinical trial investigating the safety and efficacy of using SDT to target and destroy these tumors. SDT involves using focused ultrasound to activate a non-toxic agent that then causes cell death only within the activated region. In total, 18 patients will be enrolled in the trial, which is taking place at three sites in the US.  

Preliminary data generated from the dose ranging cohort of this trial were presented at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting in Vancouver, Canada. 

Read SonALAsense’s Press Release