Brain tumour surgery aid gets orphan drug status

A drug used by brain surgeons to aid detection and removal of brain tumours has been granted orphan drug status by the TGA.

The drug, Gliolan. has been in-licensed to Melbourne biopharma Specialised Therapeutics Australia (STA). It is currently only available to neurosurgeons under the federal government's Special Access Scheme.

Gliolan, which was first developed by Germany's Medac and subsidiary Photonamic, causes cancerous tissue to glow fluorescent red during brain surgery. This helps surgeons visualise the boundary between healthy and diseased brain tissue.

The reaction is caused by Gliolan's active substance, the photoreceptive compound 5-aminolevulinic acid, being absorbed by cells and converted by enzymes into photoreceptive chemicals including protoporphyrin IX.

Glioma cells take up more of the compound and convert it more rapidly, resulting in tumour tissue glowing red when illuminated under a blue light of a specific wavelength. Healthy tissue, by contrast, appears blue.

The drug is administered three hours before surgery. To date, it has been used in four procedures in Australia, performed at the Royal Melbourne Hospital and Brisbane's Wesley Hospital.

Medac has been granted orphan drug designation for “photodynamic diagnosis of gliomas that are glioblastoma multiforme (GBM) (malignant) on preoperative imaging, and intended for gross macroscopic resection of all visible tumour.”

STA now plans to submit an application for TGA approval later in the year. The drug has already been approved for use in 29 countries, including the UK, France, Germany and Korea.

STA CEO Carlo Montagner said he expected the TGA approval process to take between 12 and 18 months. “We look forward to making this product broadly available to patients as it has been shown to significantly improve outcomes in glioma patients.”

During a phase III trial of Gliloan, complete resection of malignant brain tumour tissue was achieved in 65% of patients dosed with the drug, compared to 36% of patients in the control arm.