Metreleptin

What is Metreleptin?

• Metreleptin (MYALEPT) is a leptin analog used with a diet recommended by your healthcare provider to treat problems caused by not having enough leptin in your body (leptin deficiency).

What are the uses of this medicine?

This medicine is used with a diet recommended by your healthcare provider to treat problems caused by not having enough leptin in your body (leptin deficiency) in people with congenital or acquired generalized lipodystrophy.

Limitations of Use: It is not known if MYALEPT is safe and effective when used:

• to treat problems (complications) caused by partial lipodystrophy
• to treat liver disease, including non-alcoholic steatohepatitis (NASH)

MYALEPT should not be used to treat:

• people with HIV-related lipodystrophy
• people with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without signs or symptoms of congenital or acquired generalized lipodystrophy.

How does this medicine work?

• MYALEPT (metreleptin) for injection exerts its function by binding to and activating the human leptin receptor (ObR), which belongs to the Class I cytokine family of receptors that signals through the JAK/STAT transduction pathway.

Who Should Not Use this medicine ?

This medicine cannot be used in patients with:

• general obesity that is not caused by a congenital leptin deficiency.
• Hypersensitivity to metreleptin.

What drug interactions can this medicine cause?

• No formal drug interaction studies were performed.

Is this medicine FDA approved?

• It was approved for use in the United States in 2014.

How should this medicine be used?

Recommended Dosage:

• Body weight 40 kg or less: starting dose 0.06 mg/kg/day, increase or decrease by 0.02 mg/kg to a maximum daily dose of 0.13 mg/kg.
• Males greater than 40 kg body weight: starting dose 2.5 mg/day, increase or decrease by 1.25 mg to 2.5 mg/day to a maximum dose of 10 mg/day.
• Females greater than 40 kg body weight: starting dose 5 mg/day, increase or decrease by 1.25 mg to 2.5 mg/day to a maximum dose of 10 mg/day.

Administration

• Use MYALEPT exactly as your healthcare provider tells you to.
• Your healthcare provider will tell you how much MYALEPT to use and when to use it. Do not change your dose unless your healthcare provider tells you to.
• Do not suddenly stop using MYALEPT. Stopping MYALEPT suddenly may cause a serious problem with your pancreas (pancreatitis) and very high triglycerides.
• If your healthcare provider decides that you should stop using MYALEPT, your healthcare provider should slowly decrease your dose (taper) over 1 week.
• MYALEPT is injected 1 time a day at the same time each day.
• MYALEPT can be used with or without food.
• If you miss a dose of MYALEPT, take it as soon as you remember. Take your regular dose the next day at your normal time. Do not take more than your regular daily dose in a single day. Do not take an extra dose or increase the amount of your dose to make up for a missed dose.
• Do not mix MYALEPT and insulin in the same syringe or vial. Although MYALEPT and insulin doses may be given at the same time, do not inject MYALEPT and insulin in the same injection site.
• When MYALEPT is used in newborns or infants, your healthcare provider will tell you if MYALEPT should be mixed with a liquid called sterile water for injection (WFI) (preservative-free). For older children and adults, bacteriostatic water for injection (BWFI) may be used.
• MYALEPT is given as an injection under the skin (subcutaneous) of your stomach (abdomen), thigh, or upper arm. Do not inject MYALEPT into a vein or muscle.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As a sterile, white, solid, lyophilized cake of 11.3 mg metreleptin per vial to deliver 5 mg per mL when reconstituted in 2.2 mL of BWFI or WFI.

This medicine is available in fallowing brand namesː

• MYALEPT

What side effects can this medication cause?

The most common side effects of this medicine include:

• headache
• low blood sugar (hypoglycemia)
• decreased weight
• abdominal pain

MYALEPT may cause serious side effects, including:

• risk for loss of endogenous leptin activity or loss of MYALEPT efficacy due to neutralizing antibodies
• lymphoma (a type of blood cancer)
• low blood sugar (hypoglycemia)
• autoimmunity
• allergic reactions (hypersensitivity)
• benzyl alcohol toxicity

What special precautions should I follow?

• Anti-metreleptin antibodies with neutralizing activity Could inhibit endogenous leptin action and/or result in loss of MYALEPT efficacy. Test for neutralizing antibodies in patients with severe infections or loss of efficacy during MYALEPT treatment.
• Three cases of T-cell lymphoma have been reported in the MYALEPT lipodystrophy program. Carefully consider benefits and risks of treatment with MYALEPT in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy.
• MYALEPT is available only through a restricted distribution program under a REMS, called the MYALEPT REMS Program.
• A dose adjustment, including possible large reductions, of insulin or insulin secretagogue may be necessary. Closely monitor blood glucose in patients on concomitant insulin or insulin secretagogue therapy.
• Autoimmune disorder progression has been observed in patients treated with MYALEPT. Carefully consider benefits and risks of MYALEPT treatment in patients with autoimmune disease.
• Hypersensitivity reactions (e.g., anaphylaxis, urticaria or generalized rash) have been reported. Patient should promptly seek medical advice regarding suspected reactions.
• MYALEPT contains benzyl alcohol when reconstituted with BWFI. MYALEPT contains no preservative when reconstituted with sterile Water for Injection (WFI). Preservative-free WFI is recommended for use in neonates and infants.

What to do in case of emergency/overdose?

• Prolonged overdose was associated with severe anorexia causing vitamin and zinc deficiencies, iron deficiency anemia, protein calorie malnutrition, and poor weight gain, which resolved following supportive treatment and dose adjustment.

Management of overdosage:

• In the event of an overdose, patients should be monitored and appropriate supportive treatment be initiated as dictated by the patient's clinical status.

Can this medicine be used in pregnancy?

• Available pharmacovigilance reports with the use of MYALEPT in pregnant women are insufficient to evaluate for any drug-associated risk for major birth defects, miscarriage, or adverse maternal or fetal outcomes.
• Women who become pregnant during MYALEPT treatment are encouraged to enroll. Patients or their physicians should call 1-855-669-2537 to enroll.

Can this medicine be used in children?

• The MYALEPT study included a total of 35 pediatric patients (73%) with an age range from 1 to 17 years.
• No clinically meaningful differences were observed in the efficacy and safety of MYALEPT between pediatric and adult patients.

What are the active and inactive ingredients in this medicine?

• Active Ingredient: metreleptin
• Inactive Ingredients: glutamic acid, glycine, sucrose, and polysorbate 20

Who manufactures and distributes this medicine?

• Manufactured by:

Amryt Pharmaceuticals DAC Dublin, Ireland MYALEPT is a registered trademark and the property of the Amryt Pharma Group.

What should I know about storage and disposal of this medication?

• Store MYALEPT in the refrigerator between 36°F to 46°F (2°C to 8°C).
• KEEP MYALEPT vials in their carton and out of the light.
• Do not freeze MYALEPT.
• Do not use MYALEPT past the expiration date printed on the vial.
• Do not use MYALEPT if the white powder in the vial is discolored.
• After mixing, the MYALEPT liquid in the vial should be clear and colorless. Do not use MYALEPT if it is colored or cloudy, or has any lumps or particles in it. Throw the vial away and get a new one.
• After mixing, do not freeze or shake MYALEPT.

MYALEPT mixed with BWFI:

• MYALEPT can be used for more than 1 dose for up to 3 days when stored in the refrigerator between 36°F to 46°F (2°C to 8°C) and out of the light. Throw away any unused MYALEPT after 3 days.

MYALEPT mixed with WFI:

• Should be used right away. Throw away any unused MYALEPT, it cannot be saved for later use.

• Dailymed label info on Metreleptin
• FDA Metreleptin

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