Carthera has received an orphan medical device status endorsement for its SonoCloud ultrasound system for treating brain disorders under a new pilot programme by the European Medicines Agency (EMA).
The France-based company’s SonoCloud system delivers ultrasound to temporarily open the blood-brain barrier. Activated ahead of drug administration, the skull implant is intended to allow therapeutic agents to better reach the brain.
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Past research has shown that the blood-brain barrier, a natural filtration system of the brain, limits the penetration of more than 98% of drugs.
The endorsement means that Carthera will benefit from advice, including support around its clinical development strategy and clarification on the final steps before filing a European CE mark application for its SonoCloud system, from an EMA expert panel.
Intended for medical device manufacturers and notified bodies, the endorsement is part of an EU pilot programme to support early access pathways for innovative devices targeting rare diseases.
As part of the programme, the EMA has prioritised certain types of orphan medical devices such as those treating life-threatening medical conditions or those that could cause permanent impairment of a bodily function, and novel devices with potential major clinical benefits.
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By GlobalData“This is a very important step for the company, since it creates a unique forum for dialogue with the expert panel prior to submitting the technical documentation for conformity assessment,” said Sandra Thiollière, director of regulatory affairs at Carthera.
“In parallel, we are continuing our early-stage contacts with our notified body to prepare for CE marking submission.”
Carthera is currently evaluating SonoCloud in improving the treatment of recurrent glioblastoma (rGBM).
In 2024, the Sorbonne University spinout shared results from a Phase I/II single-arm trial (NCT03744026) assessing the safety and efficacy of blood-brain barrier disruption with an implantable ultrasound system in rGBM patients receiving carboplatin.
In 12 patients who received carboplatin just before using the device, the one-year overall survival rate (OSR) was 58%, with a median OS of 14 months from surgery. The historical rate of survival for patients living with rGBM is around 9-11 months.
Carthera is currently recruiting patients for a pivotal trial of SonoCloud. The two-arm trial will enrol 560 patients and evaluate the system combined with carboplatin (CBDCA) versus standard of care, lomustine (CCNU) or temozolomide (TMZ), in patients undergoing planned resection for first recurrence glioblastoma.
