FDA Grants Orphan Drug Designation to Opaganib in Neuroblastoma

• The FDA has granted an orphan drug designation to opaganib (Yeliva, ABC294640) for the treatment of patients with neuroblastoma.
• Neuroblastoma develops from immature nerve cells and makes up 15% of all pediatric cancer-related deaths.
• Opaganib has now received 2 orphan drug designations by the FDA, with the first being for cholangiocarcinoma treatment.

Opaganib has gained FDA orphan drug designation for the treatment of patients with neuroblastoma, a pediatric malignancy arising from neuroblasts, immature neural crest cells.¹

If opaganib is approved in neuroblastoma, this orphan drug designation offers a 7-year marketing exclusivity period. This also leads to additional benefits, including accelerated development and review times, potential grant funding, and possible tax credits.

"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," said Mark Levitt, MD, PhD, chief scientific officer at RedHill, in a press release.

Cancer cells with pink bubbles: © Adobe Contributor - stock.adobe.com

Neuroblastoma is the most common extracranial solid tumor in children, accounting for approximately 15% of pediatric cancer-related deaths. While it typically affects children aged 5 or younger, it can also occur in older children. Approximately 650 new cases of neuroblastoma are diagnosed in the US each year, and general treatment strategies include a combination of surgery, chemotherapy, radiation therapy, and sometimes targeted therapy or immunotherapy.

Opaganib is a proprietary, investigational, host-directed, and potentially broad-acting drug. The first-in-class agent is administered orally and is a sphingosine kinase-2 selective inhibitor with anticancer, anti-inflammatory, and antiviral activity. Preclinically, opaganib has shown positive results for the treatment of renal fibrosis. It also shows the ability to potentially target multiple oncology, radioprotection, viral, inflammatory, and gastrointestinal indications.

The agent is currently in development for the treatment of multiple diseases, including obesity-related syndromes, prostate cancer, cholangiocarcinoma, gastrointestinal acute radiation syndrome, Sulfur Mustard exposure, COVID-19, Ebola, and more.²

Opaganib has undergone studies in advanced cholangiocarcinoma and prostate cancer. The phase 2a clinical study (NCT03377179) of opaganib in patients with advanced, unresectable cholangiocarcinoma led to it being granted its first orphan drug designation from the FDA.

An investigator-sponsored phase 2 study (NCT04207255) evaluating opaganib in patients with prostate cancer has also completed enrollment. Patient follow-up for this study is ongoing at the Medical University of South Carolina Hollings Cancer Center and Emory University.

A phase 1 chemoradiotherapy study protocol is also ready for FDA investigational new drug submission.¹

"Opaganib has broad oncology potential with promising preliminary clinical data in solid tumor cancers such as prostate cancer and [cholangiocarcinoma], 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. We also see such utility extending to the potential for opaganib to have a sensitizing effect in hormone receptor pathway inhibition therapy, which the Company expects to test in a planned externally funded phase 2 study," added Levitt in the press release.

REFERENCES:

1. RedHill's opaganib granted orphan drug designation by the FDA for childhood cancer, neuroblastoma. News release. RedHill Biopharma Ltd. August 26, 2024. AccessedAugust 26, 2024. https://tinyurl.com/3j3snf8v

2. Pipeline. RedHill Biopharma Ltd. Accessed August 26, 2024. https://www.redhillbio.com/our-programs/pipeline/opaganib/default.aspx