Agency should require physician training, facility certification, and patient registry as a condition for approving BioMarin's and CSL Behring’s investigational gene therapies, the advocacy group says. It calls for treatment to be limited to the population studied with no-off label use.
The National Hemophilia Foundation is urging the US Food and Drug Administration to impose restrictions on the use of gene therapies for hemophilia as a condition of their approval in order to assure their safe use.
The patient advocacy group submitted a citizen petition to the agency asking it to require a Risk Evaluation and...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
