CBER’s lean into accelerated approval slashes three years from Sangamo gene therapy
FDA will accept Phase I/II data on Fabry gene therapy, obviating need for Phase III trial
FDA’s Peter Marks has been saying for some time that the agency will lean into the accelerated approval pathway to speed the development of gene therapies for rare diseases. Tuesday’s announcement that CBER, of which Marks is the director, has agreed to accept interim data on kidney function from a Phase I/II trial of Sangamo’s Fabry disease gene therapy as the basis for an accelerated approval application demonstrates how the expedited pathway can have dramatic impacts for rare disease patients and for biotechs.
CBER’s acceptance of estimated glomerular filtration rate (eGFR) slope, a measure of kidney function, as an approvable endpoint will eliminate the need for a Phase III trial, Sandy Macrae, president and CEO of Sangamo Therapeutics Inc. (NASDAQ:SGMO), told BioCentury. ...