Mydicar is a genetically targeted enzyme replacement therapy designed to restore levels of SERCA2a, that regulates calcium cycling and contractility.
The randomized double-blind placebo-controlled Phase 2 Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease (CUPID) trial is intended to investigate the efficacy and safety of Mydicar therapy.
In the study, patients treated with high dose Mydicar met its primary safety and efficacy endpoints, and reported an 88% risk reduction of major cardiovascular events.
During 12-months, the mean duration of hospitalisation in the Mydicar high dose group was 0.4 days per patient compared with 4.5 days per patient in the placebo group.
Celladon president and CEO Krisztina Zsebo said the positive results of the Phase 2 Cupid trial demonstrated the ability of Mydicar to treat patients with chronic, advanced heart failure.
"Today we are pleased that the FDA recognizes the potential benefit of Mydicar to address the enormous unmet medical need for additional therapeutics to treat advanced heart failure patients by granting the program Fast Track Status," Zsebo added.