Exon‑skipping trial shocks, Sarepta $5.5B earnings
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- Debra Miller raised $1.3 million for a Dutch scientist’s startup and helped turn CureDuchenne into a powerhouse.
- The FDA experienced an internal “civil war” over exon‑skipping drugs, and a senior official overrode reviewers to approve the first candidate.
- Sarepta Therapeutics has earned over $5.5 billion from its exon‑skipping drugs.
- Hawken Miller enrolled in a clinical trial of a new exon‑skipping drug about 2½ years ago.
- The trial of Hawken and 38 other patients produced results that have stunned some of the field’s top experts.
Why it matters: Patients with Duchenne and their families gain a tangible sign of a potentially disease‑modifying therapy, while the $5.5 billion revenue amassed by Sarepta Therapeutics from exon‑skipping drugs sits alongside expert doubts about the real benefit of those treatments. The trial’s surprising data underscore the urgent need for more robust clinical evidence.
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