Real Stories From People Living With
Generalized Lipodystrophy (GL)

Raeya

Leptin is a hormone that Raeya’s body doesn’t make enough of, and Myalept is a leptin replacement therapy, so it made sense for Raeya to start Myalept right away.

From Jilandre, Raeya’s mother:

I knew my baby was different

Raeya was born with congenital generalized liposdystrophy, even though we didn’t know it at the time. But as soon as she was born and handed to me, I knew there was something to be concerned about.

Her skin was tight—not like other newborn babies I’d seen. She was also very muscularly defined, even though her belly was round. She looked like a bodybuilder.

Things got worse

Shortly after her birth, Raeya became jaundiced, which was confusing since her appetite seemed almost insatiable. She was always hungry. Then, when she was about 2 months old, Raeya’s blood work revealed high triglycerides and high blood sugar. Basically, this tiny baby with no fat had the blood work of an obese adult.

We then saw a gastroenterologist and found out that her liver was enlarged, which explained the size of her belly. She also had advanced bone age. We went back and forth seeing probably eight different specialists. It wasn’t until a geneticist we’d seen went to a seminar about lipodystrophy that suddenly all of the questions and concerns about Raeya made sense. Finally, when she was 5 months old, she was diagnosed with congenital generalized lipodystrophy.

We were finally able to give her disease a name and get some answers. We learned that Raeya’s body doesn’t produce enough leptin. And the lack of leptin in Raeya’s body was contributing to her high triglycerides and high blood sugar.

What happened next

Giving Raeya the Myalept injection was difficult at first because of her lack of fat, but over time it got easier. The nurses trained us in how to mix the medication and give the shot, then it was our turn. It was a two-person job the first time. Raeya says she used to be afraid, but now it only hurts sometimes. In fact, now she'll walk up to me when it's time for her shot. I've encouraged her to get involved and I give her some choice about where she gets the injection--she'll put her finger right over the spot where I should do it so she feels more in control.

Raeya's day-to-day life is a lot like any other kid, but she gets a shot every morning. She's a bubbly, full-of-energy kid. Every day she gets up and lives her life like nothing is holding her back.

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.

Jilandre's daughter, Raeya, lives with GL and is taking Myalept, and Jilandre has agreed to share their personal experience with Myalept and GL. Before taking Myalept, always ask your doctor about the benefits and risks of the treatment. Individual results may vary.

Important Safety Information

What is Myalept?

Myalept^® (metreleptin) 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.

Important Safety Information

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 your 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.

Other possible side effects

The most common side effects of Myalept include headache, low blood sugar, decreased weight, and/or abdominal pain.

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).

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.

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 doctor 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 www.FDA.gov/medwatch or call 1-800-FDA-1088.

This is the most important information about Myalept. For more detailed information, please see the patient Medication Guide and Full Prescribing Information including Boxed Warning.

Important Safety Information

What is Myalept?

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.

Important Safety Information

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 your 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.

Other possible side effects

The most common side effects of Myalept include headache, low blood sugar, decreased weight, and/or abdominal pain.

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.

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.

This is the most important information about Myalept. For more detailed information, please see the patient Medication Guide and Full Prescribing Information including Boxed Warning.