Regulatory update for malignant glioma drug

MRI showing glioblastoma

Carthera, a spin-off from Sorbonne University, 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.

Carthera is using carboplatin in the first registrational trial of its SonoCloud technology in the treatment of recurrent glioblastoma (rGBM). It 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.

“We are thrilled to receive orphan drug designation from the European and US authorities. This is an important milestone for the company as it encourages us to push ahead with our pivotal trial using carboplatin to treat recurrent glioblastoma,” said Sandra Thiollière, head of regulatory affairs at Carthera.

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.

Orphan drug status

Preliminary safety and efficacy results for combining the SonoCloud-9 with carboplatin have recently been published in Nature Communications.

Carthera recently enrolled the first patients in its pivotal trial (NCT05902169) in Europe and in the US, with a projected recruitment duration of two years. Interim analysis will be conducted over the course of the study. In parallel, investigator sponsored trials with different therapeutic agents will confirm the potential of the SonoCloud platform.

The FDA’s ODD programme 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 opportunities for Carthera to extend its market exclusivity in the US and EU.

Megan Thomas, Multimedia Editor, DDW

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