Sarepta Therapeutics Comments on Erroneous Submission to US FDA Adverse Event Reporting System (FAERS)
August 08 2019 - 3:27PM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, was informed earlier today that
an adverse event report was erroneously submitted to the FDA’s
adverse event reporting system (FAERs), a post-marketing
surveillance database for approved therapies. Our
investigation to date indicates that this report was not submitted
to the FAERs database by a Sarepta employee or the study’s
principal investigator.
The submission reported a 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. He was hospitalized for
observation, discharged the following day and test results returned
to baseline.
Study 102 is a one-to-one blinded study and thus
a subject presenting an adverse event could be either 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. No stopping rule in
Study 102 was triggered.
Rhabdomyolysis is a commonly understood risk
associated with Duchenne muscular dystrophy.
About Sarepta
TherapeuticsSarepta is at the forefront of precision
genetic medicine, having built an impressive 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. The Company’s programs and research focus span
several therapeutic modalities, including RNA, gene therapy and
gene editing. Sarepta is fueled by an audacious but important
mission: to profoundly improve and extend the lives of patients
with rare genetic-based diseases. For more information, please
visit www.sarepta.com.
Internet Posting of
Information
We routinely post information that may be
important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Source: Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc.
Investors: Ian Estepan, 617-274-4052iestepan@sarepta.com
Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com
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