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The safety of PEPAXTO + dexamethasone was evaluated in 157 patients1

Non-hematologic adverse reactions with a rate of ≥10% in the HORIZON study1

Patients (N=157)
Adverse Reactions
Any Grade
Grade 3 or 4
General disorders and administration-site disorders
Fatiguea
55%
6%
Pyrexiab
24%
1.9%
Edema peripheralb
14%
1.3%
Gastrointestinal disorders
Nauseab
32%
0.6%
Diarrhea
27%
0
Constipationb
15%
0.6%
Vomiting
13%
0
Infections
Respiratory tract infectionb,c
24%
5%
Pneumoniad
13%
11%
Respiratory, thoracic, and mediastinal disorders
Cough
17%
0
Dyspnea b
11%
1.3%
Dyspnea exertional
10%
0
Metabolism and nutrition disorders
Decreased appetiteb
14%
0.6%
Hypokalemiab
14%
1.3%
Hyopocalcemiab
10%
0.6%
Nervous system disorders
Headache
13%
0
Dizziness
11%
0
Musculoskeletal and connective tissue disorders
Bone painb
13%
1.9%
Pain in extremityb
13%
1.9%
Back painb
12%
0.6%
Arthralgia
10%
0
Psychiatric disorders
Insomniab
11%
0.6%

aFatigue includes fatigue and asthenia.1

bNo Grade 4 adverse reactions occurred.1

cRespiratory tract infection includes upper respiratory tract infection, lower respiratory tract infection, respiratory tract infection, and respiratory tract infection viral.1

dPneumonia includes pneumonia, pneumocystis jiroveci pneumonia, and pneumonia viral.1

Laboratory abnormalities with a rate of ≥ 50% that worsened from baseline in the HORIZON study1

Patients (N=157)
Laboratory Abnormalities
Any Grade
Grade 3 or 4
Leukocytes decrease
99%
88%
Platelets decrease
99%
80%
Lymphocytes decrease
97%
95%
Neutrophils decrease
95%
82%
Hemoglobin decrease
84%
50%
Creatinine increasee
68%
1%

eNo Grade 4 laboratory abnormalities occurred.1

  • Grade 3/4 thrombocytopenia occurred in 43% of patients during the first cycle, with a median time to onset of 15 days from the first dose1
  • Grade 3/4 neutropenia occurred in 50% of patients during the first cycle, with a median time to onset of 15 days from the first dose1

The clinical implications of thrombocytopenia and neutropenia include:

  • Grade 3/4 thrombocytopenia with concurrent Grade 3/4 bleeding events occurred in 4 patients (3%; 1 was Grade 4)2
  • Grade 3/4 neutropenia with concurrent Grade 3/4 infections occurred in 18 patients (11%; 1 was Grade 4)2
  • Febrile neutropenia was reported in 6% of patients1

Fatal adverse reactions occurred in 10 patients (6%); none were considered related to PEPAXTO.3

In the HORIZON study, the safety profile of PEPAXTO + dexamethasone consisted primarily of hematologic adverse reactions that were managed with dose modifications and supportive care1
  • Dosage interruptions: 62% of patients, most frequently for thrombocytopenia
  • Dose reductions: 27% of patients
    • Due to thrombocytopenia in 22% of patients
    • Due to neutropenia in 6% of patients
  • Permanent discontinuation: 22% of patients

IMPORTANT SAFETY INFORMATION

PEPAXTO is contraindicated in patients with a history of serious allergic reaction to melphalan flufenamide or melphalan.

PEPAXTO may cause thrombocytopenia, which may lead to hemorrhage. Monitor platelets at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if the platelet count is less than 50 x 109/L. Withhold PEPAXTO until platelet count is 50 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Adjust dose and/or dose schedule based on signs and symptoms of bleeding.

PEPAXTO may cause neutropenia, which may lead to infection. Monitor neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if absolute neutrophil count is less than 1 x 109/L. Withhold PEPAXTO until absolute neutrophil count is 1 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Consider leukocyte growth factor as clinically appropriate.

PEPAXTO may cause anemia. Monitor red blood cell counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Treat anemia as clinically indicated. Dosage modification and dose delay of PEPAXTO may be required to allow for recovery of red blood cells.

Patients taking PEPAXTO experienced infections, including fatal infections. Consider antimicrobials as clinically appropriate.

Nonclinical safety studies with melphalan flufenamide at dosages exceeding the recommended dose for PEPAXTO were associated with mortality. The safety and efficacy of PEPAXTO has not been established for use as a conditioning regimen in patients receiving transplant.

Secondary malignancies such as myelodysplastic syndromes or acute leukemia have been reported in patients with multiple myeloma who were treated with PEPAXTO. Monitor patients long term for the development of secondary malignancies.

Based on its mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman because it is genotoxic and targets actively dividing cells. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

The most common adverse reactions (20%; Grades 1‑4) were fatigue (55%), nausea (32%), diarrhea (27%), pyrexia (24%), and respiratory tract infection (24%).

Special Considerations
Use in Pregnancy

Based on the mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman. There are no available data on PEPAXTO use in pregnant women to evaluate for a drug-associated risk. PEPAXTO is a genotoxic drug. Advise a woman of childbearing potential of the potential risks to the fetus.

Lactation

There is no information regarding the presence of melphalan flufenamide or its metabolites in human breast milk, or the effects on the breastfed child or on milk production. Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment with PEPAXTO and for 1 week after the last dose.

Females and Males of Reproductive Potential

PEPAXTO can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose.

PEPAXTO may damage the developing male germ cells, resulting in possible genetic fetal abnormalities. Advise males with a female partner of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

PEPAXTO can cause amenorrhea in premenopausal women and result in infertility. Based on findings of melphalan flufenamide in animals, PEPAXTO may impair male fertility. Alkylating drugs, such as PEPAXTO, can also cause irreversible testicular suppression in patients.

INDICATION

PEPAXTO is indicated in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38‑directed monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Limitation of Use

PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

Please click here for full Prescribing Information.

IMPORTANT SAFETY INFORMATION

PEPAXTO is contraindicated in patients with a history of serious allergic reaction to melphalan flufenamide or melphalan.

PEPAXTO may cause thrombocytopenia, which may lead to hemorrhage. Monitor platelets at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if the platelet count is less than 50 x 109/L. Withhold PEPAXTO until platelet count is 50 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Adjust dose and/or dose schedule based on signs and symptoms of bleeding.

PEPAXTO may cause neutropenia, which may lead to infection. Monitor neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if absolute neutrophil count is less than 1 x 109/L. Withhold PEPAXTO until absolute neutrophil count is 1 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Consider leukocyte growth factor as clinically appropriate.

PEPAXTO may cause anemia. Monitor red blood cell counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Treat anemia as clinically indicated. Dosage modification and dose delay of PEPAXTO may be required to allow for recovery of red blood cells.

Patients taking PEPAXTO experienced infections, including fatal infections. Consider antimicrobials as clinically appropriate.

Nonclinical safety studies with melphalan flufenamide at dosages exceeding the recommended dose for PEPAXTO were associated with mortality. The safety and efficacy of PEPAXTO has not been established for use as a conditioning regimen in patients receiving transplant.

Secondary malignancies such as myelodysplastic syndromes or acute leukemia have been reported in patients with multiple myeloma who were treated with PEPAXTO. Monitor patients long term for the development of secondary malignancies.

Based on its mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman because it is genotoxic and targets actively dividing cells. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

The most common adverse reactions (20%; Grades 1‑4) were fatigue (55%), nausea (32%), diarrhea (27%), pyrexia (24%), and respiratory tract infection (24%).

Special Considerations
Use in Pregnancy

Based on the mechanism of action, PEPAXTO can cause fetal harm when administered to a pregnant woman. There are no available data on PEPAXTO use in pregnant women to evaluate for a drug-associated risk. PEPAXTO is a genotoxic drug. Advise a woman of childbearing potential of the potential risks to the fetus.

Lactation

There is no information regarding the presence of melphalan flufenamide or its metabolites in human breast milk, or the effects on the breastfed child or on milk production. Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment with PEPAXTO and for 1 week after the last dose.

Females and Males of Reproductive Potential

PEPAXTO can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with PEPAXTO and for 6 months after the last dose.

PEPAXTO may damage the developing male germ cells, resulting in possible genetic fetal abnormalities. Advise males with a female partner of reproductive potential to use effective contraception during treatment with PEPAXTO and for 3 months after the last dose.

PEPAXTO can cause amenorrhea in premenopausal women and result in infertility. Based on findings of melphalan flufenamide in animals, PEPAXTO may impair male fertility. Alkylating drugs, such as PEPAXTO, can also cause irreversible testicular suppression in patients.

INDICATION

PEPAXTO is indicated in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38‑directed monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Limitation of Use

PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

Please click here for full Prescribing Information.

  1. REFERENCES:
  2. 1. PEPAXTO. Prescribing information. Oncopeptides, Inc.; 2021.
  3. 2. Supplement to: Richardson PG, Oriol A, Larocca A, et al. Melflufen and dexamethasone in heavily pretreated relapsed and refractory multiple myeloma. J Clin Oncol. 2020;JCO2002259. doi: 10.1200/JCO.20.02259
  4. 3. Richardson PG, Oriol A, Larocca A, et al. HORIZON (OP-106): melflufen plus dexamethasone in relapsed/refractory multiple myeloma refractory to pomalidomide and/or an anti-CD38 monoclonal antibody — primary and subgroup analysis. Poster presented at: 25th Congress of the European Hematology Association (EHA25 Virtual); June 11-21, 2020.