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Sarepta Therapeutics (SRPT +36%) is surging after announcing that the FDA approved an expansion to the labeled indication for ELEVIDYS. Sarepta, which is a leader in precision genetic medicine for rare diseases, said the FDA expanded the label to include individuals with Duchenne muscular dystrophy (DMD) who are at least 4 years of age.

• DMD is a rare genetic disease. As Sarepta explains, it predominantly affects males, but, in rare cases, can also affect females. DMD causes the muscles in the body to become weak and damaged over time and is eventually fatal. Muscle weakness becomes increasingly noticeable between the ages of 3 and 5, and most patients use a wheelchair by the time they are 12. During adolescence, heart and breathing muscles weaken, leading to life-threatening complications. Sadly, many pass away by their mid-20s from respiratory or cardiac failure.
• Today's news is two parts. First, the clinical benefits of ELEVIDYS in ambulatory patients has been confirmed, and thus traditional FDA approval has now been granted to all ambulatory Duchenne patients of 4+ years. So that was good to see. Second, the FDA feels this therapy is reasonably likely to confer clinical benefit in non-ambulatory patients. As a result, the FDA granted an accelerated approval for non-ambulatory patients (although verification in its ENVISION confirmatory trial will be needed to gain traditional approval). In addition, all age restrictions for individuals age 4 and above are removed.
• The key point is that approval now covers children whether they can walk (ambulatory) or not. The company explained that the initial approval of ELEVIDYS for ambulatory patients was a significant milestone, and the expanded indication means clinicians now have a treatment option for the vast majority of boys and young men living with Duchenne, meaning those that are not ambulatory.
• On the call, Sarepta explained that the importance of this expansion to the lives of patients can hardly be overstated. This represents the most significant advancement yet in its fight to bring a longer, better life to Duchenne patients. With this label expansion, ELEVIDYS is now potentially available to approximately 80+% of all diagnosed Duchenne patients.

Overall, investors are clearly excited about today's label expansion. Sarepta describes the commercial opportunity as "absolutely immense" and says it is well prepared for a robust launch and does not expect any supply constraints. The drug is expensive, but when compared to chronic therapy costs, which typically increase over time, the price is reasonable. The news comes just nine days after Pfizer's (PFE) DMD study did not meet its primary endpoint. Sarepta is a dominant player in the DMD space and today's news was a big win for them and DMD patients generally.