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  • Writer's pictureSaeed Anwar Anuj

Sarepta's micro-dystrophin gene therapy for DMD: an unexpected twist in the journey

I've previously navigated you through the landscape of existing therapies and introduced you to Sarepta's micro-dystrophin gene therapy, SRP-9001. This treatment, awaiting FDA approval, had lit a beacon of hope in the DMD community. Now, as the plot thickens and we find ourselves amid suspenseful anticipation, let's delve deeper into the recent developments.


On May 24, the FDA deferred its much-awaited decision on the approval of SRP-9001 from May 29 to June 22. This change, although unexpected, serves to remind us of the intricate complexities involved in drug approval processes. The FDA's mandate is to safeguard public health by ensuring our drug's safety, efficacy, and security. Therefore, a deferral, far from a negative sign, illustrates the regulator's commitment to exhaustive review and responsible decision-making.


In an interesting twist, the FDA has indicated an initial potential approval of SRP-9001 for a smaller patient group than previously expected—specifically for DMD patients aged between 4 and 5. This represents a narrower demographic than the therapy's trial participants, who were ambulatory DMD patients with an average age of around 7 years.


While such a development may seem like a setback, it's crucial to view it through the lens of the FDA's careful balancing act—weighing the urgent need for effective treatments for debilitating diseases like DMD against the fundamental mandate of ensuring patient safety. Judah Frommer, an analyst from Credit Suisse, suggests that this narrower patient group could be perceived as a short-term challenge for Sarepta. But I believe I want to see it as a strategic and cautious stride, illustrating the stepwise and adaptive approach required in the field of gene therapy, especially for rare diseases like DMD.


The narrative becomes more intriguing as we consider the FDA's evolving stance toward gene therapies. As I've noted before, Peter Marks, who heads the FDA's biological division, has been advocating for the use of disease-related biomarkers for approval of gene therapies, especially for conditions with small patient populations. SRP-9001, based on limited data, has shown potential in helping patients produce a mini version of the essential dystrophin protein.


While this doesn't provide unambiguous evidence of the therapy's benefits, it does indicate potential efficacy. It's critical to note that Sarepta is conducting a late-stage trial to further substantiate the therapy's benefit. The first glimpses of this trial's data are expected to emerge by December, adding another layer of anticipation to our story.


As we continue to journey through the twists and turns of SRP-9001's path toward potential approval, let's take a moment to recognize the significant leaps forward that have been made. The fact that a gene therapy for DMD is on the cusp of approval is itself monumental. The journey, albeit filled with uncertainties and suspense, is a testament to our relentless pursuit of progress.


As we continue to track this unfolding narrative, let's remain hopeful and patient. After all, the rollercoaster ride toward groundbreaking medical innovation is rarely straightforward, but the destination makes the journey worthwhile.



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06 June 2023

Saeed Anwar

Edmonton, Canada

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