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Celladon Gets FDA’s “Breakthrough Therapy” Status for Gene Therapy

[Corrected 4/10/14, 12:25 pm. See below.] The FDA has granted its “breakthrough therapy” designation for a gene therapy treatment in mid-stage development by San Diego’s Celladon (NASDAQ: CLDN), for patients with advanced heart failure.

The FDA notified the company in a letter sent by fax Wednesday from the Office of Cellular, Tissue and Gene Therapies, according to Fredrik Wiklund, Celladon’s vice president for corporate development and investor relations. It is the first time the FDA has given the designation to a gene therapy program, and means the development and review of Celladon’s Mydicar therapy can be expedited, Wiklund said.

Celladon sought the designation based on a long-term, follow-up study of Cupid 1, a mid-stage clinical trial that enrolled 39 patients with severe heart failure. Patients either got a placebo or a low, mid, or high dose of Mydicar through cardiac catheterization. Results from the follow-up study, released in November, confirmed initial findings that showed a dramatic, 88 percent reduction in heart failure-related hospitalizations among patients who received the highest dose of the gene therapy treatment.

Genetic Enzyme Replacement Therapy

Celladon Illustration of Gene Therapy Treatment

After three years, the patients who got the highest dose of Mydicar still showed an 82 percent reduction in episodes of worsening heart failure and hospitalizations.

“That’s what really crystallized the strength of the data,” Celladon CEO Krisztina Zsebo said Wednesday. The safety data for Mydicar also was “superb,” and shows no drug-related toxicities, Zsebo added.

The high-dose Mydicar patients also showed an improved survival rate throughout the three-year follow-up study. Heart failure represents a large, unmet need, and the mortality rate is roughly 50 percent within five years of the initial diagnosis of heart failure, according to the company.

A second clinical trial that is intended to confirm and expand on the results of Cupid 1 began in February, after enrolling 250 patients. “For Europe, this is a pivotal study,” Zsebo said. “For the U.S., it’s still to be determined whether we need to do a phase 3 trial or not.”

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