FDA grants Celladon’s Mydicar drug fast-track status

13th December 2011 (Last Updated December 13th, 2011 18:30)

The US Food and Drug Administration (FDA) has granted fast-track designation of Celladon's investigational product candidate Mydicar, which is designed to treat advanced heart failure.

The US Food and Drug Administration (FDA) has granted fast-track designation of Celladon's investigational product candidate Mydicar, which is designed to treat advanced heart failure.

Mydicar is a genetically targeted enzyme replacement therapy designed to restore levels of SERCA2a and regulate calcium cycling and contractility.

The Phase 2 Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease (CUPID) trial is a randomised double-blind placebo-controlled study designed to evaluate the efficacy and safety of Mydicar, a genetically targeted enzyme replacement therapy for advanced heart failure.

In the study, 39 patients met its primary safety and efficacy endpoints at six months for high dose of Mydicar versus the placebo, and also showed the safety and efficacy endpoints.

Patients enrolled in the study had severe forms of the disease, defined by the New York Heart Association as class III or IV heart failure, which significantly impaired the pumping function of their hearts and less than half had the normal ability to transport and utilise oxygen during exercise testing.

Following a 12-month treatment, a single infusion of Mydicar demonstrated an 88% risk reduction of major cardiovascular events, with the mean duration of hospitalisation in the Mydicar high dose group being 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 II Cupid trial demonstrated Mydicar's ability to treat patients with chronic, advanced heart failure.

"Today we are pleased that the FDA recognises the potential benefits of Mydicar in addressing the enormous unmet medical need for additional therapeutics to treat advanced heart failure patients by granting the program fast-track status," Zsebo said.

The CUPID trial has also demonstrated the favourable safety profile of Mydicar therapy, with no significant side effects.

The primary endpoints of the study included safety, worsening of heart failure leading to hospitalisation, frequency of and time to cardiac transplantation, changes in patients' ability to exercise, echocardiographic assessments, a blood test for NT-proBNP and symptoms of heart failure.

The fast-track status facilitates the development and accelerate the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.