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Fda Accelerated Approval

All articles tagged with #fda accelerated approval

health4 hours ago•3 min saved

FDA Clears First Gene Therapy for Severe LAD-I in Children

The FDA approved Kresladi (marnetegragene autotemcel), the first gene therapy for severe Leukocyte Adhesion Deficiency Type I in pediatric patients without an HLA-matched donor, using autologous gene-modified stem cells to restore ITGB2 function and CD18/CD11a on white blood cells. The treatment is given as a single IV infusion after conditioning, based on biomarker improvements in an open-label trial, with post-marketing confirmatory studies required and safety monitoring for cytopenias and infections.

via fda.gov|

#fda-accelerated-approval #gene-therapy #health

healthcare1 year ago•2 min saved

"Study: Accelerated Approval Fails for Many Cancer Drugs"

A study found that most cancer drugs granted accelerated approval by the FDA do not demonstrate clinical benefits within five years, raising concerns about patient access to unproven medications. Despite the program's intention to speed access to promising drugs, only 43% of the drugs demonstrated a clinical benefit in confirmatory trials, yet 63% were converted to regular approval. The study highlights the need for better communication of uncertainty to patients and the importance of careful explanation by doctors. Recent updates to the program give the FDA more authority to withdraw drugs when companies don't meet their commitments, aiming to streamline the process for verifying drug efficacy.

via NBC News|

#cancer-drugs #clinical-benefit #drug-efficacy