Oncopeptides AB’s appeal of the US FDA drugs center proposal to withdraw accelerated approval for the multiple myeloma drug Pepaxto (melphalan flufenamide, or melflufen) will be the first measure of the speed offered by new expedited withdrawal procedures.
How Fast Is ‘Expedited’? Pepaxto To Show Speed Of New Withdrawal Process For Accelerated Approval Drugs
For the Pepaxto appeal to be wrapped up on a faster timeline than Genentech’s dispute with the US FDA drugs center over Avastin’s breast cancer claim, CBER Director Peter Marks, the commissioner’s designee, would have to render a final decision before January 2024.
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Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.
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The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm clinical benefit.
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Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.
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