With us today is Ivan Kairatov, a biopharma expert with deep experience in research and development, to discuss the evolving story of Sarepta’s gene therapy, Elevidys. We’ll explore how new long-term data is reshaping the clinical conversation around the therapy’s effectiveness, the commercial hurdles it faces amid safety concerns, and the real-world impact on patients with Duchenne muscular dystrophy.
New long-term data suggests the benefits of Elevidys grow significantly over three years, after the therapy initially missed its primary goal at one year. How might this “rebalance the discussion” with clinicians, and what specific metrics best illustrate this compounding effect for patients and their families?
This is exactly the opportunity Sarepta’s CEO was referring to when he talked about rebalancing the discussion. Initially, the conversation was dominated by the fact that the therapy missed its main goal at the one-year mark, which created a lot of doubt. Now, with three years of data, the narrative can shift from a single, disappointing snapshot in time to a much more compelling long-term trajectory. For a clinician, the most powerful evidence is seeing the gap between treated patients and the historical control group widen so dramatically. It wasn’t just a small change; the difference on a key motor function test grew from a statistically insignificant 0.7 points at one year to a very significant 4 points at year three. This tells a powerful story of sustained and compounding benefit, not just a temporary bump, and that’s what truly matters in a progressive disease like Duchenne.
Despite positive data, sales have slowed, and some believe the market is now limited to newly diagnosed patients. What specific steps can be taken to rebuild commercial momentum? Please describe the profile of a physician or family who remains hesitant and what might convince them.
Rebuilding momentum is a significant challenge because trust has been shaken. The company can’t just rely on data; they need to focus on targeted education and reassurance. A hesitant physician is likely one who saw the initial trial miss and the subsequent safety issues and concluded the risk-benefit profile was unfavorable. They’ve probably already treated their most clear-cut, eligible patients and are now cautious about others. Similarly, a hesitant family may have heard about the liver failure cases and are terrified. For them, a statistic is abstract, but the fear is very real. To convince them, Sarepta needs to equip neurologists with clear, digestible long-term data that shows not just efficacy but also manageable safety over time. It’s about arming clinicians for those difficult conversations, showing them that the 4-point functional improvement at three years is a tangible benefit that can outweigh the known, and now better-managed, risks.
Elevidys’ use was restricted following serious safety events, including liver failure. While no new long-term safety signals have emerged, how does a clinician practically manage these known risks? Describe the monitoring protocols and crucial conversations that must occur with families before and after treatment.
Managing these risks is an active, hands-on process that begins long before the infusion. The conversation with the family has to be incredibly transparent. A clinician must lay out the potential for serious events like liver failure, explaining that while rare, they are real possibilities. It’s a discussion that balances the hope of slowing this devastating disease with the gravity of the treatment’s risks. Post-treatment, the monitoring is rigorous. This involves frequent blood tests to monitor liver enzymes and other vital functions, especially in the initial months. The physician and family are in constant communication, watching for any sign of trouble. The fact that no new safety signals have emerged in the long-term follow-up is reassuring, but it doesn’t eliminate the initial risks. It just means the safety profile is consistent and, with careful management, can be considered manageable for the right patient.
One neurologist noted that new data mirrors her clinical observations of a clear distinction between treated and untreated patients. Can you share an anecdote of how this “tangible and measurable” impact manifests in a child’s daily life, beyond what standard clinical trial endpoints might capture?
Absolutely. A clinical endpoint, like a 4-point score improvement, can feel abstract. But what that neurologist, Dr. Crystal Pound, is talking about is the real-world difference a family sees every day. Imagine two young boys with Duchenne. The untreated boy might struggle to get up from the floor, needing to use his hands to push off his legs in that classic maneuver. Over time, he might stop being able to climb stairs or run in the yard with friends. For the treated boy, that 4-point difference might mean he can still pop up from the floor without a second thought. It could be the ability to keep up during a game of tag or to walk through the grocery store without needing a wheelchair. It’s the preservation of these small, ordinary moments of childhood that are truly the most “tangible and measurable” impact for a family.
What is your forecast for the Duchenne muscular dystrophy gene therapy landscape over the next five years?
Over the next five years, I foresee a landscape defined by both advancement and complexity. We will likely see next-generation gene therapies emerge, potentially with improved safety profiles or novel delivery mechanisms that could expand eligibility to more patients. However, the commercial and access challenges that Elevidys has faced will become a critical case study for the entire field. Payers will become more sophisticated in their evaluations, demanding robust, long-term data that clearly justifies the high cost. The discussion will increasingly focus on defining which patients benefit most and establishing a sustainable economic model. Essentially, the science will continue to accelerate, but the key to success will be navigating the intricate web of clinical evidence, regulatory scrutiny, and market access to ensure these groundbreaking therapies can actually reach the children who need them.
