Andecaliximab Granted Orphan Drug Designation

On February 19, 2024, andecaliximab, a highly selective antibody inhibitor of MMP9, was granted orphan drug designation by the European Medicines Agency (EMA). Learn more 

On March 19, 2024, the U.S. Food & Drug Administration (FDA) also granted andecaliximab orphan drug designation. Learn more 

It is important to remember that requesting regulators like the EMA and FDA to grant orphan drug designation is one step in preparation for a clinical trial. Andecaliximab must go through a phase 2 clinical trial to be tested for safety and efficacy in people with FOP (adults first, then children). The EMA and FDA will then review that data and either grant approval, deny approval, or request a phase 3 trial which will provide more data. If we look at the current trials, each phase of a trial generally takes 2-4 years to complete so the review of andecaliximab as an approved treatment for FOP is likely still several years out.

MMP9 was discussed in the February 16, 2024 Journal of Bone & Mineral Research publication on findings from the "Resilient Patient Project." You can learn more about MMP9 and FOP at 





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