Product Differences Necessitate Some US FDA Inconsistency In Rare Diseases, Center Directors Say
Executive Summary
Development standards for a one-time gene therapy should be different from a drug taken regularly, CBER Director Peter Marks says.
You may also be interested in...
Impasse At US FDA Could Mean Stealth Abandons Barth Syndrome Treatment
In an interview with the Pink Sheet, Stealth’s CEO says she is not confident the company could design a clinical trial in the rare disease that would meet the FDA’s expectations.
Cell/Gene Therapies: US FDA Wants ‘Quantitative Correlation' Between Surrogate And Clinical Outcome, Former Reviewer Says
When weighing accelerated approval, the FDA wants to see that ‘X change in the surrogate translates into Y change in the clinically meaningful outcome,’ Parexel’s Steve Winitsky says. CBER Director Peter Marks opines on the types of therapeutic settings where accelerated approval is best suited, and what happens when a confirmatory trial disappoints.
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.