I included more than one scholarly article because these are all intertwined together. All of them are about a drug candidate to treat Duchenne Muscular Dystrophy (DMD) a rare genetic disorder. I could go into detail about how all of the work that came together for this drug to get FDA approval that was interdisciplinary by nature. However I’d rather talk about it in a way that relates more to me and my interest in it.
Sarepta Therapeutics is in the process of making many different drugs for the treatment of DMD. They use ground breaking new solutions to these rare genetic diseases. By skipping an exon it looks to cure people suffering of DMD. Exon skipping was for a long time in the world of science fiction and questioned of being feasible. Eteplirsen and exon 51 skipping drug skips the 51st exon and has recently shown clinical benefit doing so.
However the phase 2 study used to show efficacy of Eteplirsen and its exon skipping ability is very controversial. The placebo group that was used to compare against those who were receiving the phase 2 treatment were not really a placebo group during the time this study was done. They were a placebo group that happened over 50 years ago. In the science community this brings about a lot of controversy. A placebo group should be receiving the placebo around the same time the studied group is receiving the drug. Leaving the actual efficacy of the drug in question, especially when there was only around a 30% difference in results from the placebo group. However Eteplirsen got priority review and orphan drug designation giving Sarepta the only rights to create this drug.
To me this has meaning outside of my interest in drug development with a focus on rare disease because I was also shorting Sarepta Therapeutics stock in a stock simulator. The FDA approval of eteplirson was extremely controversial and unlikely. However now the DMD community has access to a drug that will hopefully improve the lives of thousands. Exon skipping has finally been proven doable and may give us even more understanding of hopefully soon to be a new era of genetically focused drugs as we move away from small particle. Seeing as the majority of diseases today are genetic by nature.
I and many other people spent hours creating models to show the probability of Sarepta getting eteplirsen FDA approval. As well as forecasting what their future cahsflows might look like if the drug got approved. My forecast’s were pretty close though I didn’t think eteplirsen would get approved. I estimated they would make around 300 million in revenue after the approval of eteplirsen. They exceeded that making near 400 million mostly because the patients who were receiving the treatment were older and required larger doses. This taught me a very good lesson, and in the end I was happy with the results. If people truly are getting healthier with this drug than it is one of the greatest most recent scientific breakthroughs. A lot of people right now are hoping the efficacy of eteplirsen is there. However only time will tell as Sarepta enters into their phase 3 study on eteplirsen.