FDA grants orphan drug status for RedHill’s opaganib for neuroblastoma

This designation is set to provide opaganib with seven years of marketing exclusivity upon approval for this indication.

Orphan drug status may also offer additional benefits, including potential grant funding and tax credits, alongside accelerated development and review processes.

Opaganib is an orally administered drug with a host-directed mechanism of action.

It selectively hinders sphingosine kinase-2 (SPHK2), which may confer anticancer, anti-inflammatory, and antiviral effects.

The drug is also being investigated for its potential in treating a variety of conditions, including obesity-linked syndromes, prostate cancer, and cholangiocarcinoma, as well as gastrointestinal acute radiation syndrome (GI-ARS), exposure to Sulfur Mustard, Covid-19, Ebola, and other viruses.

The host-directed action of opaganib is believed to hinder multiple cellular pathways, induce autophagy and apoptosis, and disrupt viral replication.

This is achieved through the simultaneous inhibition of three sphingolipid-metabolizing enzymes (SPHK2, DES1, and GCS) in human cells.

Opaganib is under evaluation in two US government countermeasures programmes addressing Acute Radiation Syndrome (ARS) and Sulfur Mustard exposure, both of which are supported by the National Institutes of Health (NIH).

In addition to neuroblastoma, opaganib has been granted orphan drug designation for cholangiocarcinoma and has been studied for treating advanced cholangiocarcinoma and prostate cancer.

The drug also has a ready-to-submit Phase I chemoradiotherapy study protocol for FDA Investigational New Drug (IND) application, the company noted.

Preclinical studies have shown positive results of the opaganib in renal fibrosis, indicating its potential in various oncology, radioprotection, viral, inflammatory, and gastrointestinal indications.

RedHill chief scientific officer Dr Mark Levitt said: “RedHill is proud to have received a second orphan-drug designation for opaganib in oncology, following its previous designation for cholangiocarcinoma. This designation for neuroblastoma – the most common infancy malignancy and for which new options are urgently needed – adds to opaganib’s potential as a novel oncological agent.

“Opaganib has broad oncology potential with promising preliminary clinical data in solid tumour cancers such as prostate cancer and CCA, and data from a range of US government supported and Apogee conducted preclinical studies in various indications, including radioprotection, and also in combination with RedHill’s RHB-107.”