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: [[ticker: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.”

Celladon’s gene therapy is intended to boost

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Author: Bruce V. Bigelow

In Memoriam: Our dear friend Bruce V. Bigelow passed away on June 29, 2018. He was the editor of Xconomy San Diego from 2008 to 2018. Read more about his life and work here. Bruce Bigelow joined Xconomy from the business desk of the San Diego Union-Tribune. He was a member of the team of reporters who were awarded the 2006 Pulitzer Prize in National Reporting for uncovering bribes paid to San Diego Republican Rep. Randy “Duke” Cunningham in exchange for special legislation earmarks. He also shared a 2006 award for enterprise reporting from the Society of Business Editors and Writers for “In Harm’s Way,” an article about the extraordinary casualty rate among employees working in Iraq for San Diego’s Titan Corp. He has written extensively about the 2002 corporate accounting scandal at software goliath Peregrine Systems. He also was a Gerald Loeb Award finalist and National Headline Award winner for “The Toymaker,” a 14-part chronicle of a San Diego start-up company. He takes special satisfaction, though, that the series was included in the library for nonfiction narrative journalism at the Nieman Foundation for Journalism at Harvard University. Bigelow graduated from U.C. Berkeley in 1977 with a degree in English Literature and from the Columbia University Graduate School of Journalism in 1979. Before joining the Union-Tribune in 1990, he worked for the Associated Press in Los Angeles and The Kansas City Times.