FDA SUPPORTS Benitec & Axovant

FDA update last night -

U.S. Department of Health and Human Services Food and Drug Administration Center for Biologics Evaluation and Research February 2019

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM585414.pdf

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM585417.pdf

"The following are hypothetical examples of regenerative medicine therapies that CBER may consider for breakthrough therapy designation:

In advanced forms of age-related macular degeneration, subretinal administration of retinal pigment epithelium cells is associated with substantial improvement in either visual acuity or visual fields, or a substantial reduction in the area of geographic atrophy, at one year post-administration." 

"Based on FDA’s interpretation of section 506(g), human gene therapies, including genetically modified cells, that lead to a sustained effect on cells or tissues, may meet the definition of a regenerative medicine therapy"

"We know that cell-based therapies and gene therapies may hold transformative promise for patients. This is why the FDA is so committed to encouraging and supporting safe and effective innovation in this field."