The agency will consider a submission that includes pooled data from ongoing studies, a decision analysts viewed as a notable, additional sign of regulatory flexibility for gene therapies.
According to the company, the agency has “indicated openness” to an accelerated approval filing for its treatment — a gene therapy called LX2006 for the neurodegenerative condition Friedreich’s ataxia — that’s based on pooled data from ongoing studies as well as results from a planned pivotal trial.
