pharmacopeia
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Boxed warning

EMBRYO-FETAL TOXICITY IMPAVIDO may cause fetal harm. Fetal death and teratogenicity occurred in animals administered miltefosine at doses lower than the recommended human dose. Do not administer IMPAVIDO to pregnant women. Obtain a serum or urine pregnancy test in females of reproductive potential prior to prescribing IMPAVIDO. Females of reproductive potential should be advised to use effective contraception during IMPAVIDO therapy and for 5 months after therapy [see Contraindications ( 4.1 ), Warnings and Precautions ( 5.1 ), Use in Specific Populations ( 8.1 , 8.8 ) and Nonclinical Toxicology ( 13.1 )] . WARNING: EMBRYO-FETAL TOXICITY See full prescribing information for complete boxed warning. IMPAVIDO may cause fetal harm. Fetal death and teratogenicity, occurred in animals administered miltefosine at doses lower than the recommended human dose. Do not administer IMPAVIDO to pregnant women. Obtain a serum or urine pregnancy test in females of reproductive potential prior to prescribing IMPAVIDO. Advise females of reproductive potential to use effective contraception during therapy and for 5 months after therapy ( 4.1 , 5.1 , 8.1 , 8.8 , 13.1 ).

Mechanism of action

Sourced from openFDA

Miltefosine is an anti-leishmanial agent [see Clinical Pharmacology ( 12.4 )] .

Indications

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  • IMPAVIDO (miltefosine) capsules are indicated in adults and adolescents ≥12 years of age weighing ≥ 30 kg for the treatment of: Visceral leishmaniasis caused by Leishmania donovani [see Clinical Trials ( 14.1 )] . Cutaneous leishmaniasis caused by Leishmania braziliensis , Leishmania guyanensis , and Leishmania panamensis [see Clinical Trials ( 14.2 )] .

Contraindications

Sourced from openFDA
  • Pregnancy ( 4.1 , 8.1 , 8.8 , 13.1 ). Sjögren-Larsson-Syndrome ( 4.2 , 12.3 ).contraindicated

Dosage & administration

Sourced from openFDA

The treatment duration is 28 consecutive days. Administer with food to ameliorate gastrointestinal adverse reactions. Table 1: Miltefosine Dosage Weight Dosage and Administration 30 kg to 44 kg One 50 mg capsule twice daily with food (breakfast and dinner) 45 kg or greater One 50 mg capsule three times daily with food (breakfast, lunch, and dinner) Administer with food to ameliorate gastrointestinal adverse reactions. 30 to 44 kg: one 50 mg capsule twice daily for 28 consecutive days ( 2 ). 45 kg or greater: one 50 mg capsule three times daily for 28 consecutive days ( 2 ).

Warnings & precautions

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Embryo-Fetal Toxicity. Do not use in pregnant women. Obtain a urine or serum pregnancy test prior to initiation of therapy. Advise use of effective contraception in females of reproductive potential ( Boxed Warning , 5.1 , 8.1 , 8.8 , 13.1 ). Reproductive effects. Miltefosine caused testicular atrophy and impaired fertility in male rats and impaired fertility in female rats. Advise patients of reproductive toxicities in animal studies and that the potential effects on human fertility have not been adequately evaluated ( 13.1 ). Renal Effects. Monitor serum creatinine during therapy and for 4 weeks after end of therapy ( 5.3 , 6.1 ). Hepatic Effects. Monitor transaminases and bilirubin during therapy ( 5.4 , 6.1 ). Gastrointestinal Effects. Encourage fluid intake ( 5.5 ). Thrombocytopenia. Monitor platelet count during therapy for visceral leishmaniasis ( 5.6 , 6.1 ). Absorption of Oral Contraceptives. Advise use of alternative method of contraception if vomiting and/or diarrhea occur ( 5.7 ). Stevens-Johnson syndrome. Discontinue IMPAVIDO ( 5.8 ). 5.1 Embryo-Fetal Toxicity Miltefosine may cause fetal harm. Embryo-fetal toxicity, including death and teratogenicity, was observed in animals administered miltefosine prior to mating, during early pregnancy, and during organogenesis at doses lower than the maximum recommended human dose (MRHD). Do not use IMPAVIDO in pregnant women. Obtain a urine or serum pregnancy test prior to prescribing IMPAVIDO to females of reproductive potential.

Adverse reactions

Sourced from openFDA

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Adverse reactions occurring in ≥2% of patients include nausea, vomiting, diarrhea, headache, decreased appetite, dizziness, abdominal pain, pruritus, somnolence, elevated transaminases, and elevated creatinine (6.1). To report SUSPECTED ADVERSE REACTIONS, contact Profounda, Inc. at 1-866-588-5405 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Visceral Leishmaniasis One Phase 3 trial was conducted in patients ≥ 12 years of age in India. Two-hundred and ninety-nine (299) patients (211 men and 88 women) received oral IMPAVIDO at a target dose of 2.5 mg/kg/day for 28 days (50 mg capsule once daily if weight was less than 25 kg and 50 mg capsule twice daily if weight was 25 kg or greater). Patients ranged between 12 and 64 years of age. Weight ranged between 15 and 67 kg (mean weight 38.6 kg) and BMI ranged between 8.2 and 24 (mean 16.1). Ninety-nine (99) patients received 1 mg/kg/day amphotericin B deoxycholate intravenously every other day for 15 doses. A statistically significant higher percentage of men received IMPAVIDO compared to amphotericin B. Less than 1% of patients who received IMPAVIDO died (2/299) and no patient who received amphotericin B died. Serious adverse reactions were reported in 2% of IMPAVIDO recipients (6/299) and 1% of amphotericin B recipients (1/99).

Use in specific populations

Sourced from openFDA

Pregnancy: IMPAVIDO should not be used during pregnancy. Obtain a urine or serum pregnancy test in females of reproductive potential prior to prescribing ( 4.1 , 5.1 , 8.1 , 8.8 , 13.1 ). Nursing Mothers: Discontinue drug or nursing depending on importance of drug to mother. Avoid breastfeeding for 5 months after IMPAVIDO therapy ( 8.3 ). Females and Males of Reproductive Potential: Advise females to use effective contraception during therapy and for 5 months after therapy. Advise patients of reproductive toxicities in animals, and that the potential for impaired fertility in humans has not been adequately evaluated ( 5.1 , 5.2 , 8.8 ). 8.1 Pregnancy Pregnancy Category D Risk Summary IMPAVIDO may cause fetal harm. Human pregnancy data are not available, however, embryo-fetal toxicity including death and teratogenicity, was observed in embryo-fetal studies in rats and rabbits administered oral miltefosine during organogenesis at doses that were respectively 0.06 and 0.2 times the maximum recommended human dose (MRHD), based on body surface area (BSA) comparison. Numerous visceral and skeletal fetal malformations were observed in a fertility study in female rats administered miltefosine prior to mating through day 7 of pregnancy at doses 0.3 times the MRHD. Do not administer IMPAVIDO to pregnant women.

Pharmacokinetics

Sourced from openFDA
Metabolism
The pharmacokinetic parameters of miltefosine in patients with visceral and cutaneous leishmaniasis treated for 28 days with IMPAVIDO are listed in Table 5. Due to the long half-life of miltefosine (> 6 days), trough plasma concentrations did not appear to reach a steady state at the end of treatment (i.e., Day 28).

Overdosage

Sourced from openFDA

The common adverse effects of vomiting, diarrhea, and abdominal pain are likely in case of overdose. Institute adequate hydration to prevent the risk of impaired renal function, and replace electrolytes as necessary. Because miltefosine is only slightly excreted in the urine, forced diuresis will not increase miltefosine excretion. Gastrointestinal lavage is of unknown value. A specific antidote to treat miltefosine overdose is not known.

Approval history

Sourced from openFDA
  • Mar 19, 2014NDANDA204684Knight Theraps

FAERS reports

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Reference statistics only. FAERS reports are voluntarily submitted and are not incidence rates, safety signals, or causal evidence. Counts reflect reporting volume — how often a reaction was reported, not how often it occurs. For decision-grade use, consult openFDA and the FAERS Public Dashboard directly.
372 total reports matchedLatest report Share = reports listing the reaction ÷ total matched reports. Rows can sum to >100% because a single report often lists multiple reactions.
  1. 1Off Label Use11631%
  2. 2Drug Ineffective10528%
  3. 3Drug Ineffective For Unapproved Indication5715%
  4. 4Nausea5214%
  5. 5Condition Aggravated338.9%
  6. 6Vomiting277.3%
  7. 7Product Use In Unapproved Indication267.0%
  8. 8Disease Recurrence246.5%
  9. 9Visceral Leishmaniasis205.4%
  10. 10Renal Failure174.6%
  11. 11Acute Kidney Injury164.3%
  12. 12Decreased Appetite164.3%
  13. 13Toxicity To Various Agents164.3%
  14. 14Gastrointestinal Disorder154.0%
  15. 15Acanthamoeba Infection143.8%

Clinical trials

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The 10 most recently updated of 50 ClinicalTrials.gov registrations naming Miltefosine as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

Frequently asked questions

How does Miltefosine work?
Miltefosine is an anti-leishmanial agent [see Clinical Pharmacology ( 12.4 )] .
What is Miltefosine used for?
According to FDA labeling, Miltefosine carries indications including: IMPAVIDO (miltefosine) capsules are indicated in adults and adolescents ≥12 years of age weighing ≥ 30 kg for the treatment of: Visceral leishmaniasis caused by Leishmania donovani [see Clinical Trials ( 14.1 )] . Cutaneous leishmaniasis caused by Leishmania braziliensis , Leishmania guyanensis , and Leishmania panamensis [see Clinical Trials ( 14.2 )] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
What class of drug is Miltefosine?
Miltefosine is classified as Other agents against leishmaniasis and trypanosomiasis, Antileishmanial, Lysophospholipid Receptor Interactions.
What are the brand names for Miltefosine?
Miltefosine is marketed under brand names including Impavido.
What are the contraindications for Miltefosine?
Miltefosine labeling lists contraindications including: Pregnancy ( 4.1 , 8.1 , 8.8 , 13.1 ). Sjögren-Larsson-Syndrome ( 4.2 , 12.3 ).. Always consult the full prescribing information and a clinician.
Note. Data for miltefosine is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.

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