Carthera's carboplatin receives Orphan Drug Designation for use in malignant glioma

Published: 14-May-2024

The combination of carboplatin with SonoCloud, an ultrasound technology designed to open the blood brain barrier, could show high potential for treating patients with malignant glioma

Carthera, a spin-off from Sorbonne University founded by Prof. Alexandre Carpentier, has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the use of carboplatin in the treatment of malignant glioma.

The FDA’s ODD program provides orphan status to drugs and biologics intended for the treatment, prevention or diagnosis of a rare disease or condition; those affecting less than 200,000 people in the US. The EMA’s orphan designation is targeted at conditions that affect no more than five in 10,000 people in the EU.

Both offer unique opportunities for Carthera to extend its market exclusivity in the US and EU.


Enhancing chemotherapy efficacy with SonoCloud

Carthera is using carboplatin in the first registrational trial of its SonoCloud technology — an ultrasound-based medical device to treat a wide range of brain disorders — in the treatment of recurrent glioblastoma (rGBM).

The study is a randomised, multicentre, two-arm clinical trial with a 1:1 ratio, aiming to recruit 560 patients across 40 sites in Europe and the US. The open-label, comparative pivotal trial will evaluate overall survival in patients undergoing carboplatin chemotherapy and treated with the SonoCloud-9 system to open the Blood-Brain Barrier (BBB).

This will be compared to the standard of care (lomustine and temozolomide) in patients with first recurrence of GBM. 


Why carboplatin?

Carboplatin has been chosen for its proven anti-tumoural activity and has already been tested as a monotherapy in patients with glioblastoma. It shows limited or no Central Nervous System (CNS) toxicity at high doses or in direct exposure.

However, it has limited penetration through the BBB when administered using standard intravenous (IV) routes. Brain concentrations of carboplatin can be significantly enhanced when coupled with the SonoCloud-9 system to temporarily disrupt the BBB. Preliminary safety and efficacy results for combining the SonoCloud-9 with carboplatin (NCT03744026) have recently been published in Nature Communications.

“This is an important milestone for Carthera, with both US and European authorities recognising the significance of brain treatments when combined with our SonoCloud device. We believe that our technology can help harness the therapeutic efficacy of carboplatin to enhance the treatment of glioblastoma patients,” said Frédéric Sottilini, CEO of Carthera.

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