Duchenne muscular dystrophy (DMD) is a particularly cruel genetic disease. It most commonly affects boys, who by the age of three, begin to experience muscle weakness in the legs, hips and pelvis.
DMD is progressive, and eventually, the heart and respiratory system are affected (remember, the heart is actually a muscle). Until fairly recently, patients did not survive beyond their teen years. However, better cardiac and respiratory care have improved the lives of many with DMD.
There is no cure for DMD, but a turn of events has given parents and children with this rare disease reason to hope. Last year, the FDA fast-tracked a drug called eteplirsen after extensive lobbying from advocacy groups and also parents of kids with DMD. Finally, here is a drug that can delay the onset of disability but without FDA approval, it would remain outside the reach of the community for whom it was intended.
The FDA actually listened to patients and families, and accelerated approval was granted.
You’d think all would be well in Pharmaville, but no. Several people questioned whether or not the FDA was setting a precedent for a small sub-set of people. Not only did they question the overall meaningfulness of the drug’s approval, they went so far as to suggest all “profits” be held in escrow until further trials were held.
Not to mention, Scott+Scott, Attorneys at Law, LLP recently announced an investigation into Sarepta Therapeutics’ findings in their DMD eteplirsen trial.
Personally, I think if eteplirsen had been their golden child, none of these concerns would have been raised. Clearly, if pharma starts really, truly putting the patient first, more people would benefit and timelines might just get a little shorter on the road to approval.
In this case, I am speaking only about eteplirsen. Other drugs do require multiple clinical trials–it’s just that, if eteplirsen has been proven to work for patients with DMD, why wait?
