Ramsey Baghdadi
Latest From Ramsey Baghdadi
Accelerated Approval For Gene Therapies: FDA’s Wilson Bryan Offers A ‘Framework’
Bryan, who oversees reviews of gene and cell therapies in US FDA’s biologics center, cautioned against falling in love with a biomarker when it is showing no ‘clinical effect.’
Drug Pricing And User Fees: CBER Will Need To Build Up As Gene Therapies Become More Popular
A quickened shift toward gene therapies for small populations may be the result of the new Medicare drug pricing law given protections afforded to those types of products.
US FDA’s ALS Science Strategy Includes Near-Term Rare Neurodegenerative Disease Task Force
Five-year plan includes multi-phased approach to ALS at the same time that Amylyx’s AMX0035 is pending review with a late September decision deadline; action plan’s mid-term priorities for FY 2023-2024 include a cell and gene therapies safety project.
Senate Commerce Leaders Want An FTC Study Into PBM Business Practices – And More
Committee Chair Maria Cantwell (D-Wash.) solicits advice from witnesses for additional authorities to conduct oversight of PBMs and pushes for a GAO study.
Gene Therapy Reviews Return To US FDA Advisory Committee Schedule
The upcoming review of two novel gene therapies from bluebird bio marks will be the first time the US FDA’s CTGTAC has met for a genetic product review in five years. That is a surprisingly long gap – and one that probably will not be repeated.
US FDA Oncology Center Makes Dose Optimization Personal
‘Project Optimus’ is continuing to press forward on reframing the approach to dosing of anticancer drugs. The initiative is very personal to OCE Director Richard Pazdur.