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FDA Grants Breakthrough Status to Gene Therapy for First Time

Celladon became the first company to receive breakthrough status for a gene therapy treatment in development. The treatment, MYDICAR, is intended to reduce the risk of heart failure in patients with a deficiency of the enzyme SERCA2a.

The FDA made its decision to grant breakthrough status to MYDICAR on the basis of Celladon’s Phase 1 trial of 39 systolic dysfunction patients. The study reported that subjects receiving high doses of MYDICAR experienced fewer heart failure events than subjects given a placebo, by a factor of more than 80%, and that this reduction was sustained for three years after treatment. No safety issues were reported in this initial trial. Breakthrough status, which is reserved for treatments targeting life-threatening diseases that show significant improvements over the standard of care, allows closer communication with the FDA and potentially an accelerated approval process.

BioIT World