The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Medannex's novel therapy MDX-124, for the treatment of biliary tract cancer (BTC). The FDA’s decision is based on emerging clinical data from the ongoing first-in-human Phase 1b ATTAINMENT clinical trial, which shows promising efficacy signals in patients treated with MDX-124 monotherapy. Durable disease control and tumour shrinkage have been observed in several BTC patients, with no significant side effects.