One of the most controversial drug approvals of 2016 was that of Eteplirsen, Sarepta Therapeutics Inc (Nasdaq:SRPT)’s then-lead development asset targeting Duchenne muscular dystrophy (DMD). Many reading might remember the situation. For those that don’t, the drug had demonstrated a degree of efficacy in clinical trials when measured against quality-of-life endpoints but, in what is generally regarded as the gold standard biomarker for these patients, the levels of dystrophin in the body (more on this shortly), patients that received Eteplirsen as part of the study only saw an average increase of dystrophin (in terms of percentage of normal levels from baseline) of 0.16% to 0.44%.

That’s an around 3.5 times multiple, but it is a far claim to be anywhere near a return to normal.

With that said, DMD is a devastating disease and is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births and amounting to about 20,000 new cases each year worldwide.

Before Eteplirsen, there was no approved treatment for the condition and therein lay the controversy – would the FDA approve the drug based on a tiny efficacy signal so as to give these patients something to take that could potentially alleviate their symptoms?

A well-publicized meeting took place at which the head of the FDA was questioned and interviewed by DMD sufferers and their parents (most of these patients are children) and the outcome was yes – the agency would approve the drug.

Markets expected a relatively slow uptake, however, despite this asset being the only option this market. As it turns out, things have been anything but slow and Sarepta is now expecting to bring in $125 million in revenues from the drug during 2017 (reliant on a near-term approval in Europe).

So why is this news now?

Well, the company has spent the last 12 months trying to develop what it calls a next-generation version of Eteplirsen, called golodirsen. The mechanism of action of the drug is different at its core from that of Eteplirsen, but the route it is taking to therapy is essentially the same.

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