People with GL do not have enough leptin

Lack of Fat Tissue



Generalized lipodystrophy (GL) is a rare disease that results in having little to no fat tissue all over the body. Not only does the disease affect your appearance, the lack of body fat results in a loss of leptin, an important hormone that helps the body function properly. 

Leptin is important because it helps control some key metabolic processes in the body. Not having enough leptin can lead to high triglycerides (fat in the blood) and high blood sugar. This is where Myalept®, the only FDA-approved leptin replacement therapy, may be able to help. Talk to your doctor to see if Myalept is right for you.


Lack of Fat Tissue

Leptin helps control certain important metabolic processes in the body:

Controlling how fat is broken down or stored. When fat is not used and stored properly in GL, it can collect as triglycerides in the blood.

Helping the body respond to insulin. Insulin is a hormone produced by the pancreas that helps manage levels of sugar in the blood. Without leptin, the body can develop insulin resistance, which may lead to diabetes in people with GL.

How GL and low leptin levels can affect you

People with GL have leptin deficiency and should track 3 important measures:


To track these problems, people with GL should be aware of 3 important measures:

  • Triglyceride level. This is the amount of fat in the blood.
  • Blood sugar level, also referred to as blood glucose level. This is the amount of sugar in the blood.
  • A1C level. This is a measure of the average blood sugar level over the course of 2 to 3 months. When blood sugar levels are too high, the risk of developing diabetes can increase in people with GL.

Not having enough leptin can cause these levels to be higher than normal in people with GL. Simple blood tests ordered by your doctor can show the level of triglycerides and sugars in the blood.

Because people with GL are often hungry, the extra fats and sugars taken in from food can make these high levels worse, resulting in a continuous cycle.



We’re here to help

Whether you want more information, have questions, or are looking for resources and support, we're by your side. Connect with ByMySide to learn more.

To speak with a ByMySide Coordinator, call 1-855-6MYALEPT (1-855-669-2537), Monday through Friday, 8 am to 8 pm ET.

ByMySide is a personalized support program for Myalept patients.  When you complete the Patient Authorization Form for the ByMySide program you can access additional support services and resources at no cost to you.  These are in addition to the support you are currently receiving in regard to your Myalept therapy through your dedicated nurse case manager including insurance investigation financial assistance (for eligible patients), 24/7 nurse support, nurse education and injection training, dietician support, and refill reminders. 

Risk of Neutralizing antibodies and risk of lymphoma.

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 nonprescription 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 or call 1-800-FDA-1088.

This is the most important information about Myalept. For more detailed information, Please see the Medication Guide and full Prescribing Information including Box Warning.

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) in people with generalized lipodystrophy.

  • 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.

Please see the Medication Guide and full Prescribing Information including Box Warning.


Photographs on this website feature people with GL and their caregivers.