Orphan drug status, given to drugs intended to treat diseases afflicting less than 200,000 people in the US, will entitle Xechem to various incentives, including the waiving of regulatory filing fees, access to potential grant funding for non-clinical and clinical research to generate required data for marketing approval, and seven years of marketing exclusivity for 5-HMF if approved by the FDA.
According to the company, 5-HMF is a natural product with very little, if any, toxicity, and a high affinity for sickle cell hemoglobin.
“We are pleased with the FDA's decision granting orphan drug designation to 5-HMF. This puts Xechem into a unique position where the company will have two drugs in the pipeline for treating patients with sickle cell disease,” said Dr Ramesh Pandey, CEO of Xechem.
“5-HMF is a new type of drug that interacts specifically with intracellular hemoglobin without interacting with other proteins in the body. Today there are no non-toxic drugs available on the market to treat SCD,” Dr Pandey continued.