FDA Breakthrough Therapy Designation: #37 From CBER For Gene Therapy
On April 10th, Celladon Corporation, a San Diego-based biotechnology company, announces that the FDA ‘s Center for Biologics Evaluation and Research (CBER) grants the company’s lead candidate, Mydicar, a Breakthrough Therapy Designation (BTD). The BTD is granted to Mydicar for reducing hospitalizations for Heart Failure (HF) in NYHA class III or IV chronic HF patients who are NAb negative. Mydicar is a novel, 1st-in-class therapy for chronic HF due to systolic dysfunction. Mydicar uses gene therapy to :
“ .. selectively target and restore SERCA2a enzyme levels by transferring the SERCA2a gene directly into cardiac muscle cells, which improves the heart’s ability to pump blood … uses a non-pathogenic recombinant adeno-associated virus (AAV) and is delivered directly to the heart in a routine outpatient procedure .. “.
Also, Celladon Corporation has developed a companion diagnostic to determine patients who are AAV1 NAb negative and thus are eligible for Mydicar. The company says that there are about 350,000 US HF patients who may be eligible.
Mydicar is currently in a Phase IIb CUPID 2 trial, with results expected in April 2015.
This is the 37th BTD announced by a sponsor company, the 6th BTD for 2014, and :
“… the first gene therapy reported to receive BTD from FDA’s CBER ….”.
CBER is the FDA’s Center for Biologics Evaluation and Research that regulates biological products for human use, such as allergenics, blood, devices, gene therapy, human tissues and cellular products, vaccines, and xenotransplantation products. As of 03/31/14 (reference latest BTD Statistics Chart), there are 2 CBER BTDs.
In Europe, the EMA (European Medicines Agency), has given regulatory approval to uniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD). No gene therapy has ever been approved by the FDA.
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