Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for Celladon's lead product candidate, Mydicar, for reducing hospitalisations for heart failure in NYHA class III or IV chronic heart failure patients who are NAb negative.
Subscribe to our email newsletter
The company is developing Mydicar as a new first-in-class therapy for patients with chronic heart failure due to systolic dysfunction.
The drug 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 president and CEO Krisztina Zsebo said the company is looking forward to working with the senior staff at the FDA to determine the most expeditious path to bring Mydicar to patients with advanced heart failure.
"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," Zsebo said.
"Mydicar has the potential to provide transformative disease-modifying effects with long-term benefits in heart failure patients with a single administration.
"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."
At present, the company is evaluating Mydicar in the randomized, 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 hospitalizations.
The trial is evaluating a single intracoronary infusion of Mydicar versus placebo added to a maximal, optimized heart failure regimen in patients with NYHA class III or IV symptoms of chronic heart failure due to systolic dysfunction.
About 250 patients have been randomized in the trial and the company expects to report results in April 2015.