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FDA grants breakthrough therapy designation for Mydicar
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Celladon Corp. has announced that the FDA granted breakthrough therapy designation for Mydicar, a treatment for patients with chronic HF as a result of systolic dysfunction.
According to a company press release, the novel, first-in-class genetic enzyme replacement therapy addresses a deficiency in the SERCA2a enzyme that causes the heart to pump inadequately in patients with HF. The outpatient procedure, in which a nonpathogenic recombinant adeno-associated virus is administered directly to the heart, is intended for patients with HF of NYHA class III or IV who are adeno-associated virus NAb-negative.
The company has also developed a diagnostic to identify Mydicar-eligible patients.
“This breakthrough therapy designation validates Mydicar’s unique characteristics and clinical data to date, and underscores the urgent need for new treatments for HF,” Krisztina Zsebo, PhD, president and CEO of the company, said in the release. “Mydicar has the potential to provide transformative disease-modifying effects with long-term benefits in HF patients with a single administration.”
The company is assessing the treatment in the CUPID 2 trial, a phase 2b, double blind, multinational trial in which 250 patients with NYHA class III or IV chronic HF due to systolic dysfunction have been randomly assigned to receive either a single intracoronary Mydicar infusion or placebo, in addition to an optimized regimen of HF treatment. According to the release, results from CUPID 2 are expected in April 2015.
Results from the phase 2a CUPID 1 trial, in which a single, high-dose intracoronary infusion of Mydicar was administered to 39 patients with advanced HF due to systolic dysfunction, indicated a reduction in the rate of HF-related hospitalization and recurrent and terminal CV events, as well as improvements to quality of life, symptoms and cardiac function markers predictive of survival, according to the release. The treatment was well-tolerated, with no safety concerns within a 3-year follow-up among Mydicar recipients.