Lax Approval Requirements Yield Blockbusters That Don’t Work
Summary
In a highly contentious 2016 decision, Sarepta Therapeutics (NASDAQ: SRPT) announced the FDA approval of its drug, Exondys 51 (eteplirsen), for Duchenne muscular dystrophy (DMD). The presence of parents of DMD patients shouting the benefits of the drug at the panel meeting appeared to persuade the CDER Director to overrule the scientific assessment of the BLA. So the seven-year lag in InterMune’s announcement of a Phase 2/3 program for Actimmune in IPF before its eventual failure could be seen as successfully eking out sales before an unpalatable clinical answer. Sarepta and Biogen have argued that conducting placebo-controlled confirmatory clinical trials after the FDA has already approved a product is unethical from the perspective of patients who would be in the placebo arm of such a study. Even if the FDA continues with its laissez faire attitude to drug approvals and confirmatory studies, revenue growth like clinical efficacy seems eventually to be curtailed by real-world evidence.
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