RedHill's Opaganib Receives FDA Rare Pediatric Disease Designation for Neuroblastoma
核心洞察
The FDA granted rare pediatric disease designation to RedHill's opaganib for neuroblastoma (搜索), adding to its existing orphan drug designation for the same indication.
New preclinical data presented at AACR 2026 showed opaganib destabilizes the oncogenic driver n-Myc (搜索) and enhances apoptosis as a potential add-on to chemotherapy in high-risk neuroblastoma (搜索) models.
The RPD designation provides eligibility for a Priority Review Voucher, while the orphan drug designation offers seven years of marketing exclusivity, PDUFA fee waivers, and tax credits upon approval.
RedHill Biopharma Ltd. (Nasdaq: RDHL) announced on June 9, 2026 that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to opaganib for the treatment of neuroblastoma (搜索), a cancer most commonly affecting babies and young children. This new designation adds to opaganib's existing orphan drug designation for neuroblastoma, further strengthening the regulatory incentives supporting the drug's development in this pediatric indication.
The RPD designation provides eligibility for a Priority Review Voucher (PRV), subject to certain conditions, which can be redeemed for priority review of a subsequent marketing application or sold to another sponsor. Combined with the orphan drug designation, opaganib now stands to benefit from accelerated development and review timelines, FDA Prescription Drug User Fee Act (PDUFA) application fee waivers, tax credits, and the potential for seven years of marketing exclusivity upon approval.
Preclinical Evidence and Mechanism of Action
New preclinical data presented at the 2026 American Association for Cancer Research (AACR) Annual Meeting demonstrated positive effects of opaganib as a potential add-on therapy in models of neuroblastoma (搜索) and triple-negative breast cancer (搜索) (TNBC). In high-risk neuroblastoma (HRNB) models, opaganib showed an ability to directly destabilize n-Myc (搜索), a key oncogenic driver of neuroblastoma and other solid tumors, through increased ceramide production that enhances programmed cancer cell death, or apoptosis.
"Receiving a cancer diagnosis is always distressing, but when it involves your child, it becomes profoundly devastating. There is an ongoing necessity to explore new alternatives that can augment treatment and enhance results for neuroblastoma (搜索), the most prevalent cancer in infants," said Dr. Mark Levitt, Chief Scientific Officer at RedHill. "In data from models of high-risk NB, presented at AACR 2026, we saw positive effects of opaganib as a potential add-on to chemotherapy."
Dr. Levitt further noted that the RPD designation, supported by data from neuroblastoma (搜索) and other preclinical oncology models along with a clinically demonstrated safety and tolerability profile, reinforces the company's belief that opaganib holds promise for improving outcomes in pediatric neuroblastoma. RedHill aims to advance development through ongoing discussions with Penn State University and the Beat Childhood Cancer consortium.
Opaganib (ABC294640) is a proprietary, first-in-class, orally administered sphingosine kinase-2 (SPHK2) (搜索) selective inhibitor. The drug's suggested mechanism of action, published in the journal Drug Design, Development and Therapy, is host-directed and potentially broad-acting, working through the simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells: SPHK2, DES1, and GCS. This inhibition is thought to induce autophagy and apoptosis while disrupting viral replication.
Neuroblastoma (搜索) Disease Burden
Neuroblastoma (搜索) is the most common cancer in infancy, with approximately 5,500 global pediatric cases per year in children aged 0 to 14. In the United States, the disease accounts for 10% of childhood cancers and 15% of pediatric cancer-related deaths. Around 750 children are diagnosed with neuroblastoma annually in the U.S. Approximately half of all neuroblastoma patients present with high-risk disease, which carries an overall five-year survival rate of approximately 50%.
Neuroblastoma (搜索) originates from immature nerve cells, or neuroblasts, and most often forms in the adrenal glands, though it can also arise in nerve cells in the abdomen, chest, neck, or pelvis. While the exact cause remains poorly understood, genetic mutations and abnormalities are known to play a role, with most cases occurring sporadically without a clear inherited pattern.
Clinical Development and Safety Profile
Opaganib has demonstrated its safety and tolerability profile in more than 470 participants across multiple clinical studies and expanded access use, including a large global Phase 2/3 study in hospitalized patients with moderate to severe COVID-19, published in Microorganisms. Beyond neuroblastoma (搜索), opaganib has received orphan drug designation from the FDA for cholangiocarcinoma (搜索) and is being evaluated in a Bayer-supported 80-patient placebo-controlled randomized Phase 2 study in combination with Bayer's darolutamide in men with metastatic castrate-resistant prostate cancer (mCRPC). A Phase 1 chemoradiotherapy study protocol is also ready for FDA-IND submission.
The neuroblastoma (搜索) market is expected to be valued at approximately $3.5 billion in 2032, according to Evaluate Ltd.