June 15 (Reuters) - Sarepta Therapeutics ( SRPT ) on
Sunday said there had been a second reported case of acute liver
failure resulting in death after a patient received the
company's gene therapy for a rare form of muscular dystrophy.
The patient was undergoing treatment with Sarepta's therapy
called Elevidys, which is the only gene therapy approved by the
U.S. Food and Drug Administration for Duchenne muscular
dystrophy patients aged four and above.
The first instance of death was reported in March this year,
a 16-year-old boy who died from acute liver failure months after
receiving Elevidys.
Liver damage is a known risk with Elevidys and other gene
therapies that use adeno-associated viral vectors to infuse
modified genes.
Sarepta said it is taking steps to mitigate the risk of
acute liver failure in patients by working to convene an
independent group of experts in Duchenne and liver health to
consider an enhanced immunosuppression regimen for Elevidys. It
has also temporarily suspending shipments of the therapy and
informed the FDA and global health authorities about the
incident.
Roche, which partners with Sarepta for
commercialization of the gene therapy outside the United States,
said in a separate statement on Sunday that it has paused the
dosing of Elevidys in patients following the two cases of fatal
acute liver failure.
(Reporting by Rishabh Jaiswal in Bengaluru; Editing by Lincoln
Feast.)