STAT+: Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval

Regenxbio's decision to submit its gene therapy for Duchenne muscular dystrophy for approval, despite initial setbacks, is a significant development in the field of genetic medicine. The company's move comes on the heels of the FDA's recent reconsideration of a Hunter syndrome gene therapy that was initially rejected. This shift in the regulatory landscape has implications for the future of gene therapy and the patients who rely on these treatments.

What Happened

Regenxbio announced that it will apply for accelerated approval for its Duchenne gene therapy, just a month after the company said regulators wanted to see another trial. This unexpected move has raised eyebrows in the scientific community, particularly given the FDA's recent reversal on a slew of drugs it had previously rejected.

Why It Matters

The development of effective gene therapies is crucial for patients with rare and debilitating diseases like Duchenne muscular dystrophy. Regenxbio's decision to push forward with its application, despite initial setbacks, demonstrates the company's commitment to bringing innovative treatments to market. The FDA's willingness to reconsider previously rejected therapies also signals a shift in the regulatory landscape, which could have far-reaching implications for the industry.

What Experts Say

"With the right structure, Americans will roll up their sleeves, do the science, and trust the result." — Manoush Zomorodi, author of an opinion piece on using public radio to recruit participants for a peer-reviewed study on walking breaks.

Background

The FDA's recent reversals on previously rejected therapies have raised questions about the agency's approval process. An abandoned heart failure trial offers a rare window into devices that the FDA deems "breakthrough" but never reach patients.

What Comes Next

As Regenxbio's gene therapy moves forward, the company will need to navigate the FDA's approval process. Meanwhile, researchers continue to explore innovative methods for recruiting participants and conducting studies, such as using public radio to reach a wider audience. The benefits of short walks and regular movement are also gaining attention, with studies showing that even small breaks can have a significant impact on wellbeing.