LA JOLLA, Calif., Dec. 12, 2011 /PRNewswire/ -- Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, announced today that its investigational product candidate MYDICAR® has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of advanced heart failure.
The Fast Track program of the FDA is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
"The positive results of the phase 2 CUPID Trial demonstrated the potential of MYDICAR® to become an important treatment for patients with chronic, advanced heart failure," said Krisztina Zsebo Ph.D., President and CEO of Celladon Corp. Dr. Zsebo continued, "Today we are pleased that the FDA recognizes the potential benefit of MYDICAR® to address the enormous unmet medical need for additional therapeutics to treat advanced heart failure patients by granting the program Fast Track Status."
About the CUPID Trial
The previously announced results of the phase 2 CUPID Trial met its primary safety and efficacy endpoints at 6 months for high dose MYDICAR® versus placebo. Additionally, 12 months after receiving a single infusion of MYDICAR®, patients treated with the highest dose versus placebo had an 88 percent risk reduction (Hazard Ratio = 0.12, P=0.003) of major cardiovascular events such as death, need for left ventricular assist device (LVAD) or cardiac transplant, episodes of worsening heart failure and number of heart failure-related hospitalizations.
The mean duration of hospitalization in the MYDICAR® high dose group during the 12-month period was 0.4 days per patient compared with 4.5 days per patient in the placebo group. This finding is especially noteworthy because heart failure is the leading cause of hospitalization in Americans 65 years of age and older.
Additionally, the 12-month CUPID data show that heart failure, which is a progressive disease, became stabilized in patients treated with high dose MYDICAR®: heart failure symptoms, exercise tolerance, serum biomarkers and cardiac function essentially improved or remained the same while these parameters deteriorated substantially in patients treated with placebo and concurrent optimal drug and device therapy.
The safety profile from this study was very favorable, with no significant side-effects from MYDICAR® therapy.
About Fast Track
Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious diseases and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.
Determining whether a disease is serious is a matter of judgment, but generally is based on whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.
Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially superior to existing therapy.
Any drug being developed to treat or prevent a disease with no current therapy obviously is directed at an unmet need. If there are existing therapies, a fast track drug must show some advantage over available treatment, such as:
- Showing superior effectiveness.
- Avoiding serious side effects of an available treatment.
- Improving the diagnosis of a serious disease where early diagnosis results in an improved outcome.
- Decreasing a clinically significant toxicity of an accepted treatment.
A drug that receives Fast Track designation is eligible for some or all of the following:
- More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval.
- More frequent written correspondence from FDA about such things as the design of the proposed clinical trials.
- Eligibility for Accelerated Approval, i.e., approval on an effect on a surrogate, or substitute endpoint reasonably likely to predict clinical benefit.
- Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA.
In addition, drugs that are designated as Fast Track will be eligible for priority review of the approval application. Fast Track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and make a decision within 60 days based on whether the drug fills an unmet medical need in a serious disease.
Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
MYDICAR® is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling and contractility. SERCA2a levels decline in all forms of late-stage heart failure resulting in deficient heart function. With MYDICAR®, the SERCA2a gene is delivered using a recombinant adeno-associated virus (AAV) as the vector. AAV is a naturally occurring virus not associated with any disease in humans. MYDICAR® is delivered in a single dose directly to the heart during a routine outpatient cardiac catheterization procedure, similar to an angiogram. MYDICAR® is synergistic and additive across current heart failure treatments such as ACE inhibitors, beta-blockers, sprinolactone/diuretics, and biventricular pacing devices. No treatment substitution decision is required by the treating physician.
About Heart Failure
Chronic heart failure is a leading cause of hospitalization and is expected to result in direct and indirect costs of $39.2 billion to the U.S. healthcare system in 2010. Nearly 6 million people in the U.S. have heart failure, and at least 670,000 new cases will be diagnosed this year. Heart failure leads to about 280,000 deaths annually. The most common symptoms of heart failure are shortness of breath, feeling tired and swelling in the ankles, feet, legs and sometimes the abdomen. There is no cure.
SOURCE Celladon Corporation