Clinical Trials
4
0 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
2019
Completed
2
50.0%
Recruiting
2
50.0%
No approval data available
- The U.S. FDA granted orphan drug designation to Attralus' zamubafusp alfa (AT-02) for the treatment of light chain (AL) amyloidosis, a rare and often fatal condition. - Zamubafusp alfa is a first-in-class pan-amyloid removal therapeutic designed to directly bind to and clear existing toxic amyloid deposits from organs, addressing a critical unmet need. - Current approved AL amyloidosis therapies only target light-chain production to reduce new amyloid formation, leaving no approved options for removing pre-existing amyloid fibrils. - The drug has completed a Phase 1 study and is currently in a Phase 2 open-label trial, with additional orphan designations already secured for ATTR amyloidosis from both the FDA and EMA.
- Bayer's investigational PET radiotracer I-124 evuzamitide successfully met primary endpoints for sensitivity and specificity in diagnosing cardiac amyloidosis in the Phase III REVEAL study. - The study evaluated 170 adults with suspected cardiac amyloidosis across 19 U.S. centers, demonstrating the potential for a single non-invasive diagnostic test for this underdiagnosed condition. - I-124 evuzamitide has received FDA Breakthrough Therapy Designation and Orphan Drug status, with Bayer planning regulatory discussions for approval submission. - Cardiac amyloidosis affects approximately 400,000 patients worldwide and typically requires 2-4 years from symptom onset to diagnosis due to lack of reliable diagnostic tools.
