Medannex’s first-in-class therapeutic granted FDA orphan drug designation for biliary tract cancer
Medannex Ltd today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its novel therapy MDX-124, for the treatment of biliary tract cancer (BTC).
BTC is a highly aggressive disease with a 5-year overall survival rate less than 20%. It accounts for over 170,000 deaths each year, highlighting the urgent need for novel treatments. The overarching term biliary tract cancer includes all cancers in the region of the biliary tree - cholangiocarcinoma, gallbladder cancer and ampullary cancer.
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. Medannex will present preliminary safety and activity data from the ATTAINMENT trial at the European Society for Medical Oncology (ESMO) Congress 2025 on 19 October.
Today’s announcement marks the second such designation from the FDA for MDX-124, with orphan status already granted for the treatment of pancreatic cancer. It provides Medannex with a range of regulatory and commercial incentives, including tax credits, fee exemptions and the potential for seven years of market exclusivity post-approval in the USA.
MDX-124 is the first treatment to target annexin-A1, a key regulator of the immune system. By inhibiting annexinA1, MDX-124 has the potential to deliver significant clinical benefit across a range of cancers and autoimmune diseases.
Dr Juan Valle, Chief Medical Officer of the Cholangiocarcinoma Foundation (USA), said:
Having seen the data generated in the ATTAINMENT trial we are encouraged by the potential of a new treatment option for patients and their families, particularly given the very good safety profile of MDX-124 thus far.
Ian Abercrombie, Chief Executive Officer of Medannex, commented:
This is another significant milestone in the development of Medannex and MDX-124. The FDA’s endorsement, based on a robust package of clinical and preclinical data, strengthens our position considerably. We are collaborating with several world-leading institutions in the USA, UK and globally to continue advancing this promising therapy for patients with serious unmet medical needs.
