Accelerated Approval In The US: It Really Is Rare Outside Of Oncology
Everyone knows that cancer drugs dominate use of the Accelerated Approval pathway by the US FDA. But it is remarkable just how rare a non-cancer AA is.
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Accelerated Approval Decline? Too Early To Overinterpret US FDA Trend
The US FDA is likely to end 2022 with noticeably fewer Accelerated Approvals granted than in recent years. But it is far too early to conclude that the agency is backing off the pathway – at least in spirit.
Accelerated Approvals Could Be Improved By Focusing On Benefit/Risk, Making Withdrawal Easier
Applying the expedited pathway in the context of a preliminary benefit/risk assessment, rather than focusing solely on predictive surrogate or intermediate clinical endpoints, would give the US FDA more flexibility, Friends of Cancer Research working group says in a new white paper.
With Sarepta’s Vyondys, US FDA Staff Saw Opportunity To Right Perceived Wrongs With Exondys
ODE I director Ellis Unger chafed at the notion of approving the Duchenne muscular dystrophy drug golodirsen on the same unvalidated surrogate marker, this time in the face of infection and renal toxicity concerns; OND’s Peter Stein granted Sarepta’s appeal while also calling attention to the company’s failure to timely conduct the eteplirsen confirmatory trial.