FDA allows Regenxbio fast-track approval for Duchenne gene therapy

Regenxbio has successfully received FDA approval to move forward with an accelerated approval pathway for its AFFINITY DUCHENNE trial. This significant milestone propels RGX-202, a gene therapy designed to treat Duchenne muscular dystrophy, into the next phase of its development. The FDA’s decision underscores the potential of RGX-202 as a breakthrough therapy, facilitating a faster clinical development process aimed at addressing the urgent and unmet needs of patients with Duchenne muscular dystrophy.

The upcoming trial is set to assess both microdystrophin expression and the functional improvements in 30 ambulatory patients who are aged one and older. Data from Phase I/II trials have already shown promising results, with participants experiencing functional improvements that surpass natural history benchmarks. Additionally, the trials demonstrated strong microdystrophin expression in those treated, and importantly, no serious safety issues were identified at either of the dose levels tested.

What's particularly noteworthy about RGX-202 is that it is the sole late-stage gene therapy for Duchenne muscular dystrophy that includes a unique microdystrophin construct with the CT domain. This incorporation is vital as it provides a potential new solution for dealing with this severe and progressively debilitating disease.

This progress marks a forward leap in the realm of treatments available for Duchenne muscular dystrophy, showing a potential pathway to improve the quality of life for many patients. The focus of the trial on both the expression of microdystrophin and the functional enhancements in young patients reflects a comprehensive approach to tackling the disease's core challenges.