Gene Therapies: US FDA Has Used Accelerated Approval Four Times Based On Intermediate Clinical Endpoints
Executive Summary
Three of the accelerated approvals were for CAR-T products, a class that the agency considers to be gene therapies. The FDA’s September approval of bluebird bio’s Skysona for cerebral adrenoleukodystrophy was the lone non-cancer product in the group. Pink Sheet infographic tracks the required confirmatory studies and timelines for all four products.
You may also be interested in...
FDA’s Pepaxto Decider: Peter Marks To Handle Oncopeptides’ Appeal Of Accelerated Approval Withdrawal
CBER director will decide whether the multiple myeloma drug should be withdrawn under new expedited withdrawal procedures; it's the third time FDA leaders have asked senior officials in the biologics center to help decide the fate of a CDER-regulated accelerated approval drug.
The Gene Therapy Wave Is Here
The long-talked about wave of gene therapy approvals is beginning to reveal itself.
BIO Worried CMS’ Alzheimer’s Coverage Is ‘Trial Balloon’ For Lower Accelerated Approval Reimbursement
In a Pink Sheet interview, BIO Chief Advocacy Officer Nick Shipley raised concerns that Medicare’s Alzheimer’s coverage decision is indicative of a broader agency view on accelerated approval that could limit drug reimbursement in both the public and private sector.