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Sarepta says adverse event report for DMD gene therapy erroneously submitted

Published 08/08/2019, 07:51 PM
Updated 08/08/2019, 07:56 PM
© Reuters.  Sarepta says adverse event report for DMD gene therapy erroneously submitted
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(Reuters) - Sarepta Therapeutics Inc (O:SRPT) said on Thursday it was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD).

Shares of the company plunged as much as 19% earlier after a notice from the Food and Drug Administration showed a patient in the study was reported to have developed a serious illness. The stock pared losses to close down about 7% after the company's statement.

Sarepta said its investigation indicated the report was not submitted by its employee or the study's principal investigator.

The drug safety monitoring board has recommended the study to continue after a review, the company said in a statement.

The incident, which occurred in February, involved a 7-year-old boy who developed rhabdomyolysis, a serious condition that can lead to complications such as kidney failure. The patient was hospitalized for observation and discharged the following day, with test results returning to baseline, the company said.

The illness was reported in the FDA adverse events database, which on its own cannot determine whether a drug is the cause of a problem, but can signal potential risks that warrant investigation.

The FDA told Reuters that it was looking into the matter and said it does not have additional information on it.

Sarepta said the study involves two arms with one receiving the drug and the other placebo, and the patient could have been on either.

The event is unlikely to be a major concern as there is a possibility that it could have occurred in a patient on placebo, RBC Capital Markets analyst Brian Abrahams said.

Analysts also pointed out that rhabdomyolysis is a commonly associated risk with DMD.

In June, Pfizer Inc (N:PFE), which is racing Sarepta to be first to market with a gene therapy for DMD, reported results from a tiny early study of its experimental treatment.

At the time, Pfizer said two of the six patients in its trial experienced side effects that landed them in the hospital, after which Wall Street analysts declared Sarepta was leading the charge to market.

Sarepta already has a drug to treat some types of DMD on the market.

DMD is a rare, genetic disorder that hampers muscle movement mainly in men, affecting one in every 3,500 to 5,000 males. A lack of dystrophin can cause life-threatening damage to the heart, and over time, death, often at a young age.

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