Regenxbio says it will submit its Duchenne muscular dystrophy gene therapy for FDA approval, a striking reversal from its prior stance. The decision comes barely a month after the company told investors that regulators wanted to see results from an additional trial.
The shift signals a calculated bet on a changing regulatory environment under the new administration. Accelerated approval would allow Regenxbio to bring the treatment to market based on surrogate endpoints rather than waiting for long-term efficacy data.
The company plans to file for accelerated approval, a path that requires demonstrating a reasonable likelihood of clinical benefit. If granted, the therapy could become one of the first gene therapies for Duchenne, a fatal genetic disorder affecting mostly boys.
An accelerated approval would also put Regenxbio ahead of rivals developing similar treatments. But the FDA will still need to weigh the risks of granting early access against the urgent need for therapies in a disease with no cure.
The agency previously emphasized the need for more robust evidence. It remains unclear whether the FDA's position has softened or if Regenxbio has new data to support its accelerated filing.