Surprise denial of rare pediatric disease priority review voucher could prompt calls for the agency to re-examine how it interprets the term ‘active ingredient’ for gene therapies. Bluebird asserts there are significant differences between its gene therapies for treating sickle cell and beta-thalassemia.
The US Food and Drug Administration’s denial of a rare pediatric disease priority review voucher for bluebird bio’s sickle cell treatment Lyfgenia (lovotibeglogene autotemcel, or lovo-cel) could prompt calls for the agency to re-examine how it interprets the term “active ingredient” in the context of gene therapies.
The FDA appears to have concluded that what it previously described as “modest manufacturing differences” between Lyfgenia and bluebird’s Zynteglo...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
