Sarepta Therapeutics Confronts Their Erroneous Submission to US FDA Adverse Reporting System

Aug 10, 2019 | FDA Adverse Reporting System

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Sarepta Therapeutics (SRPT) recently erroneously submitted to the FDA’s adverse event reporting system (FAERs), a post-marketing surveillance database for approved therapies. Upon an internal investigation, they report that the report was not submitted to the FAERs database by a Sarepta employee or the specific clinical trial’s principal investigator.

What Happened?

Someone submitted a reported case of rhabdomyolysis in a participant in Sarepta’s Study SRP-9001-102, a blinded, placebo-controlled trial investigating the use of Sarepta’s micro-dystrophin gene therapy candidate in patients with Duchenne muscular dystrophy. Two weeks post-infusion, the patient presented with dark-colored urine and elevated creatine phosphokinase (CK) levels but was otherwise asymptomatic. The patient was hospitalized for observation, discharged the following day and test results were returned to baseline.

The Study

Study 102 is a one-to-one blinded study and thus a subject presenting an adverse event could either be on active therapy or in the placebo arm of the trial. While Sarepta and its principal investigator remain blinded to the study, the study drug safety monitoring board is unblinded to the event and has reviewed the issue and recommended the study continue uninterrupted. Sarepta Therapeutics’ press release reports that no stopping rule in Study 102 was triggered.

What is Rhabdomyolysis?

A commonly understood risk associated with Duchenne muscular dystrophy. “Rhabdomyolysis occurs in about 26,000 people in the U.S. every year.” 

Who is Sarepta Therapeutics?

They are position to be at the forefront of precision genetic medicine, having developed a robust and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development.

Finances

Founded in 1980 in the Boston area, they operate at a loss of over $500 million on nearly $350 million of revenue. With a healthy market capitalization as of writing at $9 billion, capital raising options are available.

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