The FDA has granted Orphan Drug Designation to Novita Pharmaceuticals for its investigational small-molecule fascin inhibitor, NP-G2-044, for the treatment of pancreatic cancer.
NP-G2-044 has shown efficacy in blocking metastasis in preclinical and clinical studies. When combined with immune checkpoint inhibitors, it has been effective at reinvigorating anti-tumor immune responses. The drug received Orphan Drug Designation because it treats pancreatic cancer in immune checkpoint inhibitor (ICI) therapy, which is within the scope of approved orphan indication.
There are currently limited effective treatments for pancreatic cancer, especially for advanced stages. The disease has a five-year survival rate of just 12%, making it one of the deadliest malignancies.