July 21 (Reuters) - Children's Hospital Los Angeles,
citing recent U.S. Food and Drug Administration actions, on
Monday said it has paused usage of Sarepta Therapeutics' ( SRPT )
gene therapy Elevidys in all patients with muscular
dystrophy.
The FDA on Friday asked Sarepta to voluntarily halt
shipments of the gene therapy after a muscular dystrophy patient
who received a different, experimental treatment died. The
company said it did not plan to comply with the request and
would continue making the treatment available to ambulatory
patients.
The company in June suspended use of Elevidys for patients
whose disease had progressed to the point where they need a
wheelchair after two teenagers died from liver failure after
treatment.
Elevidys has full FDA approval for children with Duchenne
Muscular Dystrophy who are still able to walk, but was given
conditional approval for non-ambulatory patients.
Children's Hospital LA said its pause, effective July 18,
has been communicated to "affected patient families while it
awaits any further determination by the FDA."
The hospital is one of more than 70 authorized to administer
Elevidys. Five other major children's hospitals asked by Reuters
to comment on whether they planned to continue use of the gene
therapy did not immediately respond.
Shares of Sarepta, down about 90% year-to-date, fell 5% on
Monday to close at $13.32 on Nasdaq.