The Food and Drug Administration recently approved eteplirsen, the first drug available for use against Duchenne muscular dystrophy, the debilitating muscle wasting disease affecting young boys. The drug works through exon-skipping and is only applicable to certain forms of the disease. Currently, similar methods of gene therapy are also being developed against other forms of the disease.
Expert advisors to the FDA had voted against approving the drug, citing the relevant drug study’s small size (12 boys) and inadequate control group, which they claimed provided insufficient evidence of the drug’s efficacy. A decision on the drug’s approval was delayed for months, and the FDA, despite granting approval, is requiring additional confirmatory efficacy trials. The drug’s ultimate approval is seen by many as the direct result of an impassioned lobbying campaign by patients and their advocates. (Such advocacy groups for muscular dystrophy have historically been highly vocal and organized.) Further, with the approval of this drug, its cost has skyrocketed, adding another layer of complexity to the ethical issues surrounding this case. Many see this drug’s approval as setting a precedent for the influence of patient groups and pharmaceutical companies on medicine, with widespread disagreement over whether this is a good or a bad development.