FDA Grants US Orphan Designation to Medannex First-in-Class Therapy

Released: Wednesday 20th September 2023

FDA Grants US Orphan Designation to Medannex’s First-in-Class Therapy


Medannex Ltd (Edinburgh) announced today that its novel therapy, MDX-124, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA).


MDX-124, a first-in-class agent, is currently being studied in humans for the first time, in a Phase 1b clinical trial (the ATTAINMENT trial) in UK patients with advanced cancer.


Driven by compelling non-clinical data, the FDA designation qualifies Medannex for regulatory incentives including tax credits and fee exemptions, as well as the potential for seven years of market exclusivity post-approval in the USA.


MDX-124 is the first therapeutic agent to target annexin-A1, an important modulator of the immune system in health and disease. Targeting annexin-A1 potentially offers significant clinical benefit in the treatment of many cancers - including triple-negative breast, colorectal and lung - as well as autoimmune diseases such as multiple sclerosis, lupus and rheumatoid arthritis. The Orphan Drug Designation has been granted for the treatment of pancreatic cancer, which is the third most common cause of cancer-related death in the USA (American Cancer Society, 2022) and therefore represents a significant unmet clinical need.


Earlier this year, MDX-124 was awarded Orphan Medicinal Product positive opinion from the European Medicines Agency (EMA) as well as an Innovation Passport as part of the Innovative Licencing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (MHRA).


Medannex CEO Ian Abercrombie said: ‘This is another significant milestone in the evolution of Medannex and MDX-124. We are delighted to receive such an endorsement from the FDA based on a very strong non-clinical package. This will strengthen our position in the world’s largest healthcare market and we look forward to commencing our clinical programme within the USA’.



About Medannex Ltd: Medannex is a clinical-stage Scottish biopharmaceutical company headquartered in Edinburgh. The company is developing novel treatments for various cancers, autoimmune diseases and other conditions: https://medannex.org


About MDX-124: MDX-124 is a humanised monoclonal antibody, and the first agent to specifically target and inhibit annexin-A1, a protein known to play a key role in the development of cancers, autoimmune diseases and other conditions. A first in human clinical study (‘ATTAINMENT’) is underway.


About the ‘ATTAINMENT’ clinical study: The ‘ATTAINMENT’ study is a modular, multi-arm, first in human study to evaluate the safety and tolerability of MDX-124, alone and in combination with anti-cancer treatments, in participants with locally advanced, unresectable or metastatic solid malignancies.



Medannex Ltd enquiries – email jamesingham@medannex.org



View all News