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Full Prescribing Information  |  Med Guide/IFU

Full Prescribing Information

Med Guide/IFU

Important Safety Information

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Revuforj® (revumenib) Logo

Revuforj is a menin inhibitor indicated for the treatment of relapsed or refractory acute leukemia with a
lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older.

Efficacy

Established complete remission with Revuforj1

21

%

achieved
CR + CRh

(primary endpoint)
(n=22/104)
(95% CI: 13.8, 30.3)

  • The efficacy of Revuforj was evaluated in a difficult-to-treat patient population consisting of 104 adult and pediatric patients with relapsed/refractory acute leukemia with a KMT2A translocation

  • At baseline, patients had a received a median of 2 previous regimens (range: 1–11), with 44% (46/104) having received at least 1 stem cell transplant

  • The primary study endpoint was rate of complete remission (CR) + complete remission with partial hematologic recovery (CRh)

Median time to CR + CRh

1.9

MONTHS

(range: 0.9–5.6 months)

Median duration of CR + CRh

6.4

MONTHS

(95% CI: 2.7–not estimable)

Median follow-up was 5.7 months
(range: 0.3–28.9 months)

CR + CRh
was achieved

across subgroups1:

  • 21% of patients with AML (n=18/86)
  • 19% of patients with ALL (n=3/16)
  • 50% of patients with MPAL (n=1/2)

Rate of transplant following Revuforj1

Image of a yellow line
Image of a yellow line IV bag IV bag

23

%

 (n=24/104)

of patients
proceeded
to HSCT

(n=24/104)

  • ALL=acute lymphoblastic leukemia; AML=acute myeloid leukemia; CI=confidence interval; HSCT=hematopoietic stem cell transplant; KMT2A=lysine methyltransferase 2A; MPAL=mixed-phenotype acute leukemia.

AUGMENT-101:
Designed to study a critical unmet need

As the first pivotal trial of its kind, AUGMENT-101 assessed the efficacy and safety of Revuforj in adult and pediatric patients with relapsed/refractory acute leukemia with a KMT2A translocation (AML, ALL, and MPAL)

AUGMENT-101:
an open-label, multicohort, multicenter, phase 1/2 clinical trial1,2

A total of 135 patients received Revuforj

  • 77% were adults (n=104)

  • 23% were pediatric (n=31)

Patients weighing ≥40 kg: Dose ≈ 160 mg

Patients weighing <40 kg: Dose based on BSA

Administered orally twice daily with a strong CYP3A4 inhibitor until disease progression, unacceptable toxicity, failure to achieve morphological leukemia-free state (MLFS) by 4 cycles of treatment, or hematopoietic stem cell transplantation (HSCT)

  • Patients were able to proceed to transplant while in remission and remain on study

    • 24 patients underwent HSCT following treatment with Revuforj

Key inclusion criteria1,2

  • At least 30 days of age

  • Relapsed or refractory active acute leukemia harboring a KMT2A translocation

    • Bone marrow blasts ≥5% or reappearance of blasts in peripheral blood

    • KMT2A translocation was determined by cytogenetic testing, such as FISH

    • Patients with an 11q23 partial tandem duplication were excluded

  • White blood cell count below 25,000/μL at time of enrollment

  • ECOG performance status score 0-2 or Karnofsky/Lansky score ≥50

Primary endpoints1

  • Rate of complete remission (CR) + CR with partial hematologic recovery (CRh)

  • Short- and long-term safety and tolerability

ALL=acute lymphoblastic leukemia; AML=acute myeloid leukemia; BSA=body surface area; CYP3A4=cytochrome P450 3A4; ECOG=Eastern Cooperative Oncology Group; FISH=fluorescence in situ hybridization; KMT2A=lysine methyltransferase 2A; MPAL=mixed-phenotype acute leukemia.

NCCN

RECOMMENDED

Targeted
treatment
option

National Comprehensive Cancer Network® (NCCN®)

Revumenib (Revuforj) is an NCCN Category 2A recommended treatment option for certain types of relapsed/refractory AML, adult ALL, and pediatric ALL. This makes it a first-in-class menin inhibitor in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®).3-5

NCCN makes no warranties of any kind whatsoever regarding their content, use, or application and disclaims any responsibility for their application or use in any way.

Revuforj was studied in a heavily pretreated patient population with challenging disease characteristics1

44

%

had received at
least 1 HSCT

26

%

had relapsed at
least twice

59

%

were refractory
relapse

Patient Demographics (N=104)

Median Age (years) (Range)

37 (1, 79)

Age, n (%)

<17 years old

25 (24)

≥17 years old

79 (76)

Sex, n (%)

Male

37 (36)

Female

67 (64)

Race, n (%)

Black or African American

8 (8)

Asian

10 (10)

White

75 (72)

Multiple

1 (1)

Unknown

10 (10)

Ethnicity, n (%)

Hispanic or Latino

23 (22)

Not Hispanic or Latino

76 (73)

Unknown

5 (5)

Disease Characteristics (N=104)

Leukemia morphological type, n (%)

AML

86 (83)

ALL

16 (15)

MPAL

2 (2)

Translocations,* n (%)

t(9;11)

23 (22)

t(11;19)

20 (19)

t(6;11)

10 (10)

t(10;11)

10 (10)

t(4;11)

7 (7)

t(1;11)

3 (3)

t(11;17)

2 (2)

t(11;22)

2 (2)

t(11;16)

1 (1)

KMT2A fusion partner unknown

26 (25)

Disease status, n (%)

Primary refractory

22 (21)

Untreated relapse

21 (20)

Refractory relapse

61 (59)

Prior treatment

Median no. of previous
regimens (min, max)

2 (1, 11)

Prior stem cell
transplantation, n (%)

46 (44)

Number of prior relapses, n (%)

0

22 (21)

1

55 (53)

2

20 (19)

≥3

7 (7)

One patient did not have a translocation type reported.

Revuforj Logo

Revuforj can be used
as early as first relapse

Median Age (years) (Range)

37 (1, 79)

Age, n (%)

<17 years old

25 (24)

≥17 years old

79 (76)

Sex, n (%)

Male

37 (36)

Female

67 (64)

Race, n (%)

Black or African American

8 (8)

Asian

10 (10)

White

75 (72)

Multiple

1 (1)

Unknown

10 (10)

Ethnicity, n (%)

Hispanic or Latino

23 (22)

Not Hispanic or Latino

76 (73)

Unknown

5 (5)

Leukemia morphological type, n (%)

AML

86 (83)

ALL

16 (15)

MPAL

2 (2)

Translocations,* n (%)

t(9;11)

23 (22)

t(11;19)

20 (19)

t(6;11)

10 (10)

t(10;11)

10 (10)

t(4;11)

7 (7)

t(1;11)

3 (3)

t(11;17)

2 (2)

t(11;22)

2 (2)

t(11;16)

1 (1)

KMT2A fusion partner unknown

26 (25)

Disease status, n (%)

Primary refractory

22 (21)

Untreated relapse

21 (20)

Refractory relapse

61 (59)

Prior treatment

Median no. of previous
regimens (min, max)

2 (1, 11)

Prior stem cell
transplantation, n (%)

46 (44)

Number of prior relapses, n (%)

0

22 (21)

1

55 (53)

2

20 (19)

≥3

7 (7)

One patient did not have a translocation type reported.

Revuforj Logo

Revuforj can be
used as early as
first relapse

NEXT: SAFETY

ALL=acute lymphoblastic leukemia;
AML=acute myeloid leukemia;
HSCT=hematopoietic stem cell transplant;
KMT2A=lysine methyltransferase 2A; MPAL=mixed phenotype acute leukemia.

References: 1. Revuforj® [Prescribing Information]. Syndax Pharmaceuticals, Inc.; November 2024. 2. ClinicalTrials.gov identifier: NCT04065399. https://
clinicaltrials.gov/study/
NCT04065399. Accessed May 21, 2025. 3. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology
(NCCN Guidelines®) for Acute Myeloid Leukemia V.2.2025. © National Comprehensive Cancer Network, Inc. 2025. All rights reserved. Accessed May 21,
2025. To view the most recent and complete version of the guideline, go online to NCCN.org. 4. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia V.1.2025. © National Comprehensive Cancer Network, Inc. 2025. All rights reserved. Accessed May 21, 2025. To view the most recent and complete version of the guideline, go online to NCCN.org. 5. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Pediatric Acute Lymphoblastic Leukemia V.3.2025. © National Comprehensive Cancer Network, Inc. 2025. All rights reserved. Accessed May 21, 2025. To view the most recent and complete version of the guideline, go online to NCCN.org.

IMPORTANT SAFETY
INFORMATION AND INDICATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, and/or hypotension. In clinical trials, DS occurred in 39 (29%) of 135 patients treated with Revuforj. DS was Grade 3 or 4 in 13% of patients and fatal in one. The median time to onset was 10 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.


Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10-mg IV every 12 hours in adults or dexamethasone 0.25-mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.


QTc interval prolongation: In the clinical trials, QTc interval prolongation was reported as an adverse reaction in 39 (29%) of 135 patients treated with Revuforj. QTc interval prolongation was Grade 3 in 12% of patients. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 8%, and the increase from baseline QTcF was greater than 60 msec in 18%. Revuforj dose reduction was required for 5% of patients due to QTc interval prolongation. QTc prolongation occurred in 16% of the 31 patients less than 17 years old, 33% of the 88 patients 17 years to less than 65 years old, and in 50% of the 16 patients 65 years or older.


Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

  • Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
  • Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
  • Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia

Embryo-fetal toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.


ADVERSE REACTIONS

Fatal adverse reactions occurred in 4 (3%) patients who received Revuforj, including 2 with differentiation syndrome, 1 with hemorrhage, and 1 with sudden death.


Serious adverse reactions were reported in 99 (73%) patients. The most frequent serious adverse reactions (≥5%) were infection (24%), febrile neutropenia (19%), bacterial infection (17%), differentiation syndrome (12%), hemorrhage (9%), and thrombosis (5%).


The most common adverse reactions (≥20%) including laboratory abnormalities, were hemorrhage (53%), nausea (51%), phosphate increased (50%), musculoskeletal pain (42%), infection (41%), aspartate aminotransferase increased (37%), febrile neutropenia (35%), alanine aminotransferase increased (33%), parathyroid hormone intact increased (33%), bacterial infection (31%), diarrhea (30%), differentiation syndrome (29%), electrocardiogram QT prolonged (29%), phosphate decreased (25%), triglycerides increased (25%), potassium decreased (24%), decreased appetite (24%), constipation (23%), edema (23%), viral infection (23%), fatigue (22%), and alkaline phosphatase increased (21%).


DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

  • Strong CYP3A4 inhibitors: reduce Revuforj dose

  • Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj

  • QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec.
    Restart Revuforj after the QTc interval returns to ≤480 msec


SPECIAL POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.


Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.


Pediatric: monitor bone growth and development in pediatric patients.


Geriatric: compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.


Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.


To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.


INDICATION

Revuforj® (revumenib) is a menin inhibitor indicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older.


Please see Full Prescribing Information, including BOXED WARNING.

IMPORTANT SAFETY
INFORMATION AND INDICATION

IMPORTANT SAFETY
INFORMATION AND INDICATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.