Celladon Gets Breakthrough Therapy Designation For Mydicar From FDA

(RTTNews) - Celladon Corp. (CLDN) Thursday said it has been granted breakthrough therapy designation by the U.S. Food and Drug Administration for Mydicar, a novel, first-in-class therapy for patients with chronic heart failure due to systolic dysfunction.

The therapy is to reduce hospitalizations for heart failure in NYHA class III or IV chronic heart failure patients who are NAb negative.

Mydicar uses genetic enzyme replacement therapy to correct the deficiency in the enzyme SERCA2a that becomes deficient in heart failure patients and leads to inadequate pumping of the heart.

Celladon - a clinical stage biotechnology company focused on treatment of heart failure and other diseases characterized SERCA enzyme deficiencies - has developed a companion diagnostic to identify the patients who are AAV1 NAb negative and therefore eligible for Mydicar treatment.

The company is currently evaluating Mydicar in a randomized, double-blind, placebo-controlled, multinational Phase 2b CUPID 2 trial.

Celladon said patient enrollment has been completed and 250 patients have been randomized in this trial, results of which may be announced in April 2015.