Novel Drug & Emergent Therapeutics | Pharma |  Oncology | Leukaemia

FDA approves revumenib for relapsed or refractory acute leukaemia with a KMT2A translocation
 
 Time to read: 01:29 minutes

Published on MedED: 18 November 2024
Originally published: 15 November 2024
Source: FDA Press Release

Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDR001
Category: Oncology
Crossreference: Haematology
Category tags: leukaemia, gene therapy, refractory acute leukaemia

 

 

Product Category        Product Name Company Status

Pharmaceuticals

Revuforj 
(revumenib)

Syndax Pharmaceuticals, Inc

FDA Approved

15 November 2024, FDA Press Release
 
On 15 November 2024, the U.S. Food and Drug Administration (FDA) approved revumenib (Revuforj) from Syndax Pharmaceuticals, Inc. as the first menin inhibitor for the treatment of relapsed or refractory (R/R) acute leukaemia with lysine methyltransferase 2A gene (KMT2A) translocation in adult and paediatric patients aged one year and older.
 
This landmark approval, achieved under the FDA’s Real-Time Oncology Review (RTOR) programme, follows earlier designations of Breakthrough Therapy, Fast Track, and Priority Review, reflecting its potential to address a significant unmet clinical need.
 
KMT2A-rearranged (KMT2Ar) acute leukaemia is an aggressive malignancy associated with poor prognosis and high relapse rates. Over 95% of patients with KMT2Ar leukaemia have KMT2A translocations, chromosomal rearrangements that drive the disease. More than half of these patients relapse after frontline therapies, with a median overall survival (OS) of less than one year. Outcomes with third-line or later treatments are dismal, with complete remission achieved in only 5% of cases and a median OS of under three months.
 
The efficacy of revumenib was evaluated in a single-arm cohort of the open-label, multicentre AUGMENT-101 trial (SNDX-5613-0700; NCT04065399), involving 104 adult and paediatric patients with R/R acute leukaemia and confirmed KMT2A translocations. 
 
The trial demonstrated a complete remission (CR) or CR with partial haematologic recovery (CRh) rate of 21.2% (95% CI: 13.8, 30.3), with a median CR+CRh duration of 6.4 months (95% CI: 2.7, not estimable).

Among transfusion-dependent patients at baseline, 14% achieved transfusion independence, while 48% of those initially independent maintained this status during follow-up.

 
Adverse reactions observed in ≥20% of patients included haemorrhage, nausea, infections, musculoskeletal pain, febrile neutropenia, differentiation syndrome, and laboratory abnormalities such as elevated liver enzymes and QT prolongation.
 
The approval of revumenib represents a significant advancement in the management of KMT2Ar leukaemia. It offers a novel mechanism of action for a disease with limited treatment options and poor outcomes.


Clinical Trials & Supporting Information
 

Clinical Study State         ClinicalTrial.gov Registration Study Name

Phase 2

NCT04065399

A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation (AUGMENT-101)

 


Access Original Press Release

15 November 2024 | FDA Press Release | FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation


Access Company Press Release

15 November 2024 |  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

 



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