New Jersey-based Xechem International's new treatment for sickle cell disease (SCD) has been designated an orphan drug by the FDA, the company announced.
The drug, 5-HMF, is a five-membered heterocyclic anti-sickling compound that interacts specifically with intracellular hemoglobin without affecting other proteins in the body. It produces little or no toxicity, according to Xechem.
Xechem said it intends to submit the investigational new drug application for 5-HMF in the next several months.
Orphan drug status, given to treatments for diseases that affect fewer than 200,000 people in the U.S., provides certain incentives such as a waiver of filing fees, grants for clinical trials and seven years of marketing exclusivity.