FDA Approves Syndax’s First-in-Class Menin Inhibitor with Black-Box Warning; Updates on GSK’s ADC Deal
The FDA approved Syndax Pharmaceuticals’ Revuforj (revumenib) as the first menin inhibitor for adults and children (≥1 year) with relapsed or refractory acute leukemia with a KMT2A gene translocation1356.
Revuforj is a twice-daily oral pill and represents the first drug in its class; it is indicated for acute leukemia with KMT2A rearrangement, which is associated with poor prognosis and high relapse rates135.
The approval was based on data showing a complete remission rate of just over 20%, with remissions lasting about six months; some patients proceeded to stem cell transplant after treatment158.
The drug’s labeling includes a black-box warning for differentiation syndrome, a potentially fatal reaction seen in 29% of treated patients, characterized by fever, fluid accumulation, and organ dysfunction151314.
The FDA’s decision was granted more than a month ahead of the target date and through the Real Time Oncology Review program, underscoring the urgency and importance of addressing this patient need56.
Revuforj is priced at approximately $475,000 per year prior to rebates or discounts, and will be available in different dose formulations based on patient weight1.
This is Syndax’s second major FDA approval in 2024, following their earlier approval for Niktimvo (axatilimab) in chronic graft-versus-host disease2.
GSK's ADC (antibody-drug conjugate) deal is noted as a significant industry move in oncology this period, although primary news coverage is focused on Syndax’s approval and no additional new details on the GSK ADC deal are highlighted in these results.
Sources:
1. https://www.biopharmadive.com/news/syndax-fda-approval-leukemia-drug-revuforj/733209/
2. https://www.fiercepharma.com/pharma/syndax-scores-fda-approval-acute-leukemia-drug-after-it-disappoints-another-indication
3. https://medcitynews.com/2024/11/leukemia-syndax-pharmaceuticals-fda-approval-revuforj-menin-inhibitor-sndx/
5. https://www.prnewswire.com/news-releases/syndax-announces-fda-approval-of-revuforj-revumenib-the-first-and-only-menin-inhibitor-to-treat-adult-and-pediatric-patients-with-relapsed-or-refractory-acute-leukemia-with-a-kmt2a-translocation-302307513.html
6. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-leukemia-kmt2a-translocation
8. https://bloodcancerunited.org/news/fda-approves-first-new-class-drugs-treat-advanced-acute-leukemia
13. https://antonhealth.com/fda-approves-oral-tx-for-r-r-acute-lukemia-revuforj/
14. https://www.pharmacytimes.com/view/in-a-nod-to-precision-oncology-the-fda-approves-revumenib