Product Differences Necessitate Some US FDA Inconsistency In Rare Diseases, Center Directors Say
Development standards for a one-time gene therapy should be different from a drug taken regularly, CBER Director Peter Marks says.
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Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
Ultra-Rare Orphan Drugs: Advocates Begin Laying Groundwork For New Incentive, Special Pathway
Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
Rare Disease Patients: Placebo Controls, Sham Surgeries Not Appropriate For Gene Therapy Trials
Advocates also worry that long follow-up times could prevent participation in future clinical trials.