Released on January 9, 2012 

Recently, Celladon, a biopharmaceutical company focusing on the research and development of drugs for cardiovascular diseases, announced that its drug Mydicar used in the treatment of heart failure has obtained the fast appraisal qualification of FDA, implying that the company can submit to FDA or supplement the data on the clinical studies of the drug at any time, without queuing for examination.

The fast appraisal channel enables FDA to accelerate understanding whether the drugs or biological products used in the treatment of severe diseases have any serious or life-threatening adverse effect, so as to judge whether such drugs or biological products can meet the potential medical demand. One of the important effects of the fast appraisal policy is to strengthen the early communication between FDA and the R& D company, so as to jointly increase the efficiency of successful development of new products.

The Phase II clinical study called CUPID indicated that Mydicar would become an important drug used in the treatment of chronic advanced heart failure. In the 6-month study, the drug achieved the key endpoints, with positive safety.

In addition, in the 12-month study of follow-up visit after the single-dose injection of the drug, the risk of severe cardiovascular adverse effects in the active treatment group was reduced by 88% compared to that in the placebo control group. These severe adverse effects included death, needing left ventricular assisted device or heart transplantation, exacerbation of heart failure, and re-admission due to heart disease.

Furthermore, in the 12-month study, the average lengths of stay of the high-dose Mydicar patients and the placebo control patients were 0.4 day and 4.5 days respectively. Most of the patients hospitalized due to heart failure were 65 years old or above.

Heart failure is a kind of progressive disease. The drug can effectively control the symptoms of heart failure. Clinical studies indicated that the drug could fundamentally change the exercise tolerance, serological biomarker and cardiac function of the patients, and by contrast, the above parameters of the patients in the control group worsened. In addition, in the clinical studies, there was no significant adverse effect of Mydicar.

Mydicar is a gene targeting enzyme alternative treatment drug, which can restore the level of SERCA2a protein, regulate the calcium cycle and increase the cardiac contractibility. (Bio Valley, Bioon.com)