What is Myalept?
Myalept (metreleptin), a prescription medication, is a leptin replacement therapy used with a doctor-recommended diet to treat problems caused by not having enough leptin (leptin deficiency), a condition that causes severe obesity beginning in the first few months of life. Affected individuals are of normal weight at birth, but they are constantly hungry and quickly gain weight. The extreme hunger leads to chronic excessive eating (hyperphagia) and obesity.
It is not known if Myalept is safe and effective when used to treat problems (complications) caused by partial lipodystrophy or to treat liver disease, including non-alcoholic steatohepatitis (NASH).
Myalept should not be used to treat people with HIV-related lipodystrophy or people with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without signs or symptoms of congenital or acquired generalized lipodystrophy.
How do I take Myalept?
Always take medications exactly as instructed by your doctor. Below are some important things to keep in mind while taking Myalept. Patients self-administer Myalept once a day and typically use 14 vials per month.
DO review the complete Instructions for Use before each injection
DO inject Myalept once a day, at the same time each day
DO inject Myalept at room temperature
DO mix Myalept with the right liquid depending on age
DO inject Myalept in the upper arm, thigh, or abdomen
DO store Myalept in the refrigerator between 36°F and 46°F (2°C and 8°C) and away from light
DO follow the diet recommended by your doctor
DO NOT inject Myalept into your muscle or veins
DO NOT take more than your regular daily dosage 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
DO NOT shake Myalept when mixing
DO NOT freeze Myalept
DO NOT stop taking Myalept without consulting your doctor
DO NOT use if the liquid is cloudy or colored or has particles after mixing
Also taking insulin and/or a sulfonylurea drug?
Although Myalept and insulin dose may be taken at the same time, do not inject Myalept and insulin at the same injection site. Use separate syringes for Myalept and insulin. Once you start taking Myalept, your doctor may adjust your dose of insulin and/or sulfonylurea medication to help reduce the risk of low blood sugar (hypoglycemia).
Important Safety Information
Myalept is only available through a restricted program called the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Your doctor must be enrolled and certified in the program in order to prescribe Myalept.
Myalept may cause serious side effects, including:
• risk for developing certain proteins called neutralizing antibodies that may reduce how well your own leptin or Myalept works. Side effects of these antibodies may include infection, problems with blood sugar (including diabetes), or an increase in triglycerides
• increased risk of a type of blood cancer called lymphoma
You should not take Myalept if you:
• have general obesity not caused by a congenital leptin deficiency
• are allergic to metreleptin or any of the ingredients in Myalept. Symptoms of an allergic reaction include rash, itching (hives), swelling of face, lips, tongue, or throat, problems breathing or swallowing, fainting or dizziness, rapid heartbeat.
Before using Myalept, tell you doctor if you have any medical conditions including if you:
• have or have had problems with your blood cells, including low blood cell counts (especially your white blood cells), bone marrow, immune system, pancreas, swollen lymph nodes, lymphoma, high blood triglyceride levels, or use insulin or a sulfonylurea
• are pregnant or plan to become pregnant as it is unknown if Myalept will harm your unborn baby. If you become pregnant while using Myalept, talk to your healthcare provider about registering with a program to collect information about the outcomes of moms and babies exposed to Myalept during pregnancy. You can enroll in the Myalept program by calling 1-855-669-2537
• are nursing or plan to nurse. You should not nurse while you take Myalept
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Other possible side effects
The most common side effects of Myalept include headache, low blood sugar, decreased weight, and/or abdominal pain.
For newborns and infants, mix Myalept with sterile water for injection (preservative-free) (WFI). Serious side effects including death have happened in newborns or infants who have received the preservative benzyl alcohol. Bacteriostatic water for injection contains benzyl alcohol and it should not be used to mix Myalept for newborns and infants.
You may get low blood sugar (hypoglycemia) if you take Myalept with other medicines used to lower blood sugar, such as insulin or sulfonylurea. Your doses of these medications may need to be lowered while you use Myalept. Tell your doctor right away if you experience shakiness, sweating, headache, drowsiness, weakness, dizziness, confusion, irritability, hunger, fast heartbeat, or a jittery feeling because these may be signs of low blood sugar (hypoglycemia).
Myalept may worsen symptoms caused by certain problems in your immune system (autoimmune disorder). Ask your doctor about what symptoms you should watch for that may require further testing.
Talk to your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of Myalept. For more information, speak to your doctor or pharmacist. Tell your doctor about all the medications you take, including prescription and non prescription medicines, vitamins, and herbal supplements. Take Myalept exactly as your doctor instructs you.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatch or call 1-800-FDA-1088. Please see the Medication Guide and full Prescribing Information including Box Warning.
Why is Myalept so expensive?
Myalept is so expensive because there are no generic versions of this drug. It is the only treatment available to treat leptin deficiency, there are no other cost-saving alternatives. Myalept costs $74,159 for a monthly supply, bringing the yearly price tag to $889,904. Myalept is the fourth most expensive pharmacy drug in the U.S., Amryt, raised its price by 9.9% in January this year.