Regeneron announced FDA and EMA review acceptance for cemdisiran in generalized myasthenia gravis and FDA priority review acceptance for garetosmab in fibrodysplasia ossificans progressiva. The garetosmab BLA is supported by Phase 3 OPTIMA trial data showing 94% and 90% reductions in new bone lesions.
Regeneron said the Phase 3 NIMBLE trial of cemdisiran in generalized myasthenia gravis met primary and key secondary endpoints at week 24. The company said a U.S. regulatory application has been submitted and EU filings are planned for 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07154745 |
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently |
RECRUITING | PHASE3 |
| NCT06541704 |
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy |
RECRUITING | PHASE3 |
| NCT05744921 |
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works |
RECRUITING | PHASE3 |
| NCT05070858 |
A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis |
ACTIVE_NOT_RECRUITING | PHASE3 |
