Navigation Links
Celladon Corporation Receives FDA Fast Track Designation for Its Investigational Agent MYDICAR® for the Treatment of Heart Failure
Date:12/12/2011

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.

About MYDICAR®

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
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Celladon Corporation Announces MYDICAR® Enzyme Replacement Therapy for Advanced Heart Failure Provides Sustained Reduction in Clinical Events in Patients for 18 Months Compared with Placebo
2. Celladon Provides MYDICAR(R) Program Update of First-In-Human Trial for Advanced Heart Failure at American Society of Gene Therapy Annual Meeting
3. Celladon Corporation Announces Presentation of First-in-Human Gene Transfer to Treat Heart Failure at the 12th Annual Meeting of the American Society of Gene Therapy
4. Celladon Announces Presentation of Clinical Data From First-in-Human MYDICAR(R) Trial for Advanced Heart Failure at American Heart Association Scientific Sessions
5. Leatt Corporation Announces 2011 Annual Shareholders Meeting
6. Angeion Corporation to Report Fourth Quarter and Fiscal Year 2011 Financial Results on Friday, December 16, 2011
7. Radient Pharmaceuticals Corporation Receives $500,000 in New Capital
8. Radient Pharmaceuticals Corporation Completes Restructurings with Institutional Investors to Limit Conversions and Stock Sales and Provide for Potential Additional Capital
9. China Cord Blood Corporation Reports Financial Results for the Second Quarter and First Half of Fiscal 2012
10. Ansell Limited Acquires a Minority Share in Yulex Corporation
11. The Female Health Companys Distributor, Sekunjalo Investments Corporation (PTY) Ltd., Receives Order for 5 Million FC2 Female Condoms From the Republic of South Africa Department of Health
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/24/2016)... June 24, 2016  Collagen Matrix, Inc., ("Collagen ... and manufacturing of collagen and mineral based medical ... that Bill Messer has joined the ... further leverage the growing portfolio of oral surgery, ... Bill joins the Collagen Matrix executive team ...
(Date:6/24/2016)... 2016 Research and Markets ... Electronics 2015-2025: Applications, Technologies, Forecasts" report to ... In-Mold Electronics, Smart Skin, Structural Health Monitoring, Composite ... Structural electronics involves electronic and/or electrical components and ... dumb structures such as vehicle bodies or conformally ...
(Date:6/24/2016)... DUBLIN , June 24, 2016 ... "The World Market for Companion Diagnostic Tests" report to ... World Market for Companion Diagnostics The World ... diagnostic and personalized medicine diagnostics. Market analysis in the report ... Diagnostics Test Market (In Vitro Diagnostic Kits) by Region (N. ...
Breaking Medicine Technology:
(Date:6/25/2016)... D.C. (PRWEB) , ... June 25, 2016 , ... ... discuss health policy issues and applications at AcademyHealth’s Annual Research Meeting June 26-28, ... their work on several important health care topics including advance care planning, healthcare ...
(Date:6/25/2016)... Beach, CA (PRWEB) , ... June 25, 2016 , ... ... UCLA with Magna Cum Laude and his M.D from the David Geffen School of ... Diego and returned to Los Angeles to complete his fellowship in hematology/oncology at the ...
(Date:6/24/2016)... ... 24, 2016 , ... Those who have experienced traumatic events may suffer from ... avenues, such as drug or alcohol abuse, as a coping mechanism. To avoid this ... coping following a traumatic event. , Trauma sufferers tend to feel a range of ...
(Date:6/24/2016)... ... June 24, 2016 , ... ... for accelerated orthodontic treatment. Dr. Cheng has extensive experience with all areas of ... AcceleDent, and accelerated osteogenic orthodontics. , Micro-osteoperforation is a revolutionary adjunct to ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Haute Beauty ... Barry M. Weintraub as a prominent plastic surgeon and the network’s newest partner. ... and the most handsome men, look naturally attractive. Plastic surgery should be invisible.” ...
Breaking Medicine News(10 mins):