FDA Grants “Breakthrough Therapy” Status for Gene Therapy to reduce Systolic Heart Failure

Celladon Corporation (Nasdaq:CLDN), a clinical-stage biotechnology company developing novel therapies for patients with heart failure and other diseases characterized by SERCA enzyme deficiencies, announced that its lead product candidate, MYDICAR®, has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) for reducing hospitalizations for heart failure in NYHA class III or IV chronic heart failure patients who are NAb negative. This designation is intended to expedite the development and review of drugs for serious or life-threatening conditions and where preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Celladon is developing MYDICAR as a novel, first-in-class therapy for patients with chronic heart failure due to systolic dysfunction. MYDICAR uses genetic enzyme replacement therapy to correct the deficiency in the enzyme SERCA2a, which is an enzyme that becomes deficient in heart failure patients and results in inadequate pumping of the heart. Celladon has developed a companion diagnostic to identify the patients who are AAV1 NAb negative and therefore eligible for MYDICAR treatment.

MYDICAR® is Genetically-Targeted Enzyme Replacement Therapy for Advanced Heart Failure. The goal of the MYDICAR® treatment is to restore the enzyme SERCA2a, which is deficient in heart failure, to normal levels.


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