It appears that Oncopeptides AB will be the first drug sponsor to test the US Food and Drug Administration’s new expedited procedures for accelerated approval withdrawal as it appeals an agency request to pull the new drug application for Pepaxto (melphalan flufenamide, or melflufen).
Accelerated Approval: Oncopeptides' Pepaxto Will Test New Expedited Withdrawal Process
Company’s appeal of the US FDA’s formal request to voluntarily withdraw accelerated approval of the multiple myeloma agent is expected to follow the streamlined procedures created in last year’s omnibus legislation, which would preclude a second advisory committee review of melflufen’s fate.

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The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.
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.
The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm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.
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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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.
The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm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.
A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.