STAT+: A decade ago, these drugs tore apart the FDA. Today, they might be some patients’ best hope
Why it matters: This development could offer a life-altering treatment for thousands of Duchenne muscular dystrophy patients, potentially validating two decades of advocacy and research.
- Debra Miller spent two decades advocating and fundraising, turning her group CureDuchenne into a powerhouse, to advance exon-skipping therapies for her son Hawken's Duchenne muscular dystrophy.
- Exon-skipping drugs initially caused a "civil war" within the FDA, with a top official overriding reviewers to approve early candidates, leading to over $5.5 billion in revenue for companies like Sarepta Therapeutics despite questions about their benefit.
- Hawken Miller recently enrolled in a clinical trial for a new exon-skipping drug, with results from him and 38 other patients reportedly "stunning" experts, offering renewed hope for a therapy that was long out of reach.
- STAT News highlights the broader context of pharmaceutical news, including the FDA seeking new powers over DTC ads and the EU's pledge to fight AMR, indicating ongoing regulatory and public health discussions alongside drug development.
A decade after igniting a "civil war" within the FDA, exon-skipping drugs, once controversial, are now offering a beacon of hope for Duchenne muscular dystrophy patients like Hawken Miller. His mother, Debra, tirelessly advocated and fundraised for two decades, transforming her group CureDuchenne into a powerhouse, despite the initial skepticism and the mixed efficacy of early approvals that generated billions for companies like Sarepta Therapeutics.
