Clinical-stage biotechnology firm Celladon has received breakthrough therapy designation from the US Food and Drug Administration (FDA) for its lead product candidate, Mydicar, for reducing hospitalisations for heart failure in NYHA class III or IV chronic heart failure patients who are NAb negative.
Mydicar is being developed by the company as a new first-in-class therapy for patients with chronic heart failure due to systolic dysfunction.
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The drug uses genetic enzyme replacement therapy to correct the deficiency in SERCA2a, an enzyme that becomes deficient in heart failure patients and results in inadequate pumping of the heart.
The company has developed a companion diagnostic to identify patients who are AAV1 NAb negative, making them eligible for Mydicar treatment.
Celladon president and CEO Krisztina Zsebo said: "This breakthrough therapy designation validates Mydicar’s unique characteristics and clinical data to date and underscores the urgent need for new treatments for heart failure.
"Mydicar has the potential to provide transformative disease-modifying effects with long-term benefits in heart failure patients with a single administration.
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By GlobalData"Our goal is to bring Mydicar to market as quickly as possible in the United States, where we estimate approximately 350,000 heart failure patients with currently limited remaining treatment options could be eligible for therapy."
Currently, the company is assessing Mydicar in the randomised, double-blind, placebo-controlled, multinational Phase IIb CUPID 2 trial to determine its efficacy in reducing the frequency of and/or delaying heart failure-related hospitalisations.
The trial is assessing a single intracoronary infusion of Mydicar versus placebo added to a maximal, optimised heart failure regimen in patients with NYHA class III or IV symptoms of chronic heart failure due to systolic dysfunction.
A total of 250 patients have been randomised in the trial and the company expects to report results in April 2015.
