Sarepta Learns Why Earlier GMP Control Is Better For Promising Gene Therapies
When firms are developing highly anticipated gene therapies, it’s important to establish GMP-controlled manufacturing processes earlier in development, as Sarepta discovered when US FDA placed a clinical hold on a promising Duchenne muscular dystrophy gene therapy.
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Use of historical controls and alternative study designs are allowed, but randomized control trial is preferred.
Sarepta will face a US FDA staff with which it does not have a controversial history, the Center for Biologics Evaluation and Research, likely avoiding the reviewers that evaluated its Duchenne muscular dystrophy treatment Exondys 51.