A question that I’ve often heard asked of the biotechnology industry by critics is which is more important to the CEOs, lives or dollars?
But that’s a topic for another day, because right now, both are on the line.
In one of the biggest and most important battles that biotech has ever seen, two companies are fighting it out for supremacy in the approval of a drug that could potential help sufferers of a disease for which there currently is no effective treatment…
Duchenne muscular dystrophy, or DMD, is an inherited muscle-wasting and fatal disease. In the United States, it affects somewhere between 15,000 and 20,000 boys. That’s not even mentioning the similar number of afflicted in Europe, and the thousands of others elsewhere in the world.
DMD normally puts the afflicted in wheelchairs by the teenage years and usually leads to death between the ages of 20 and 30 by way of lung and cardiac problems.
It’s a horrible disease made even more horrible by the fact that there’s currently no effective treatment.
DMD has been called the biggest focus in the biotech industry right now, and that’s because the stakes are so high for two companies seeking approval for drugs that could slow the disease.
The two companies at the forefront of this issue are Sarepta (SRPT) and BioMarin (BMRN).
And as you can imagine, there’s been a lot of patient pressure to find an answer to DMD.
But what these companies and any investors alike are also interested in is the potential $3 billion market for a successful answer for DMD.
Right now both SRPT and BMRN are seeking accelerated approval for their respective drugs, which means they would be cleared for commercial sales before the normally required level of clinical and biomedical data because they’re targeting a serious or life-threatening disease for which there currently is no effective treatment.
And it seems BMRN has just taken a significant blow in this fight, as FDA reviewers made some highly critical remarks about the company’s drug. That led to a huge jump in share price for SRPT (about 30%).
But many see SRPT as the underdog in this fight as compared to the much larger BMRN.
This battle is far from over, but whether you’re more interested in the life-saving potential or the investment opportunity of these drugs, this is a situation worth paying close attention to.
