生产企业：RedHill Biopharma Ltd.
Opaganib (ABC294640) is a first-in-class, orally administered sphingosine kinase-2 (SPHK2) selective inhibitor developed by RedHill Biopharma, with potential broad-acting anticancer, anti-inflammatory, antiviral, and metabolic activities. It has received FDA Orphan Drug designation for neuroblastoma and cholangiocarcinoma, and FDA Rare Pediatric Disease designation for neuroblastoma.
Opaganib (ABC294640) is a first-in-class, orally administered sphingosine kinase-2 (SPHK2) selective inhibitor developed by RedHill Biopharma, with potential broad-acting anticancer, anti-inflammatory, antiviral, and metabolic activities. It has received FDA Orphan Drug designation for neuroblastoma and cholangiocarcinoma, and FDA Rare Pediatric Disease designation for neuroblastoma.
Opaganib selectively inhibits sphingosine kinase-2 (SK2). This inhibition blocks the synthesis of sphingosine 1-phosphate (S1P) and its activities (which includes regulation of fundamental biological processes such as cell proliferation, migration, immune cell trafficking, angiogenesis, immune-modulation, and suppression of innate immune responses from T-cells). This drug has dual anti-inflammatory and antiviral activity targeting a host cell component and is unaffected by viral mutation, contributing to minimization of the likelihood of resistance. It is currently being investigated against COVID-19 as it has demonstrated anti-SARS-CoV-2 activity in studies.
Opaganib is an investigational drug not yet approved for commercial distribution. It has received FDA Orphan Drug designation for neuroblastoma and cholangiocarcinoma, and FDA Rare Pediatric Disease (RPD) designation for neuroblastoma. The RPD designation provides eligibility for a Priority Review Voucher (PRV). The orphan drug designation provides potential benefits including seven years of marketing exclusivity upon approval, accelerated development and review times, PDUFA application fee waivers, and tax credits. Opaganib is being evaluated in a Phase 2 study for metastatic castrate-resistant prostate cancer (mCRPC) in combination with darolutamide, and has a Phase 1 chemoradiotherapy study protocol ready for FDA-IND submission. It has also been studied in a Phase 2/3 trial for hospitalized COVID-19 patients.
共 6 项相关疾病
