Although there were no cases of hematological malignancy reported and no evidence of insertional oncogenesis in patients treated with beti-cel, FDA raises a theoretical risk due to delayed platelet engraftment, as well as malignancies seen in other lentiviral vector programs.
A US Food and Drug Administration advisory committee will consider recommendations for pretreatment screening and postmarketing safety monitoring to address the potential risk of hematologic malignancies with bluebird bio’s betibeglogene autotemcel (beti-cel), a lentiviral vector-based gene therapy for beta-thalassemia.
In a briefing document released ahead of the Cellular, Tissue and Gene Therapies Advisory Committee meeting on 10 June,...
An abbreviated licensure pathway for biologics was created by the 2009 Biologics Price Competition and Innovation Act. Biologics demonstrated to be biosimilar to or interchangeable with FDA-approved products can use the 351(k) biologics license application pathway. Biosimilars BLAs submitted under the 351(k) pathway have a 12 month user fee goal for FDA action. Updated weekly.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
New marketing authorization applications filed with the European Medicines Agency's human medicines committee (the CHMP) for review under the EU's centralized procedure. Updated monthly.
New marketing authorization applications filed with the European Medicines Agency's human medicines committee (the CHMP) for review under the EU's centralized procedure. Updated monthly.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
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