Sarepta's Expanded FDA Approval For Elevidys Potentially Shaping Future FDA Reviews, Analyst Says

On Thursday, the FDA approved the labeled indication for Sarepta Therapeutics Inc’s SRPT Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene and are at least four years old.

In June last year, the FDA granted accelerated approval to Sarepta Therapeutics’s one-time gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients aged 4 through 5 with a confirmed mutation in the DMD gene.

William Blair writes that with the label expanded, rapid uptake of Elevidys will be anticipated in the near future, leading to substantial revenue growth for Sarepta in the coming years as they address the newly indicated population.

Payers have been supportive over the past year, allowing boys to start Elevidys therapy before aging out of the previous age-restricted label. However, with the new label covering a larger population, the analyst says it will monitor if payers maintain this support, considering the potential budget impact of a large influx of patients in the near term.

William Blair reiterates the Outperform rating with a fair value estimate of $210 per share.

Thursday’s approval and label expansions mark a significant milestone for Sarepta’s DMD franchise and revenue growth.

This achievement validates Sarepta’s gene therapy platform, which aims to advance multiple programs for various subtypes of limb-girdle muscular dystrophy (LGMD).

The most advanced program, SRP-9003 (bidridistrogene xeboparvovec) for LGMD2E/R4, is currently enrolling participants for the Phase 3 EMERGENE study.

The approval’s long-term impact will likely influence the FDA and the gene therapy field, promoting a more patient-focused and cooperative FDA review process, particularly for severe diseases with limited treatment options.

William Blair says, “The lasting impact of the approval will likely shape the FDA and gene therapy space for some time.”

Price Action: SRPT shares are up 31.8% at $162.71 at last check Friday.

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