FDA answers urgent call for DMD gene therapy despite controversy, consequences

The landmark accelerated approval of Elevidys from Sarepta to treat Duchenne muscular dystrophy is a major milestone and beacon of hope for patients and families, but it’s also an approval that’s bound to shine light on the healthcare system’s lack of readiness for delivering new gene therapies to patients — an issue that will continue to impact launches for companies and timely access to gene therapies for patients. 

The decision to grant accelerated approval, particularly for a narrow age window, will also have consequences for the patients who currently are in the eligibility window, on future gene therapy development, and on confidence in the scientific rigor of FDA decision-making. ...