June 20 (Reuters) - The U.S. Food and Drug
Administration allowed the expanded use of Sarepta Therapeutics' ( SRPT )
gene therapy for all patients with Duchenne muscular
dystrophy aged four and above on Thursday.
The agency gave traditional approval to patients four
years and above who can walk, as well as accelerated approval
for those who cannot. The company, however, added that the
therapy, Elevidys, should not be used in patients with certain
mutations in the DMD gene.
The FDA gave accelerated approval to the therapy - the
first of its kind for DMD in patients between four and five
years who can walk - in June last year. Accelerated approval is
a process that allows the agency to greenlight treatments before
confirmatory data shows they work.
Continued approval for the use of the therapy in Duchenne
patients who cannot walk may be contingent on the verification
of clinical benefit in a confirmatory trial.