Sarepta Therapeutics Shares Collapse on Serious Duchenne Gene Therapy Complication

Shares of Sarepta Therapeutics fell as much as 13% on Thursday after Wall Street learned that a child participating in a trial of the company’s experimental gene therapy for Duchenne muscular dystrophy had a serious health complication.

The company had been testing a treatment for Duchenne muscular dystrophy that would insert a functioning gene into patients with the disease, who have a non-working copy of the gene.

An FDA database reported that a 7-year-old trial participant was hospitalized with a potentially life-threatening condition that can lead to kidney failure

Shares saw a 13% drop before Sarepta stopped trading its stock.

In a public statement, Sarept stated that the hospitalization was “erroneously” submitted to the FDA by someone outside of the company.

According to the company, the clinical trial has been designed so that neither the subject nor the company are aware if they’re receiving the gene therapy or a placebo. The statement also said that the health complication the boy experienced is one commonly associated with Duchenne.

CEO Doug Ingram said on the company’s earnings call Wednesday that the company had “no evidence of significant adverse or serious adverse events.”

A company spokesperson remarked on Ingram’s comments and said “the study review board … reviewed the event and recommended the trial continue. No stopping rule in Study 102 was triggered.” The spokeswoman also said the event was “reported through the appropriate clinical trial protocol.”

Disclaimer: We have no position in Sarepta Therapeutics Inc. (NASDAQ: SRPT) and have not been compensated for this article.