Real Stories From People Living With
Generalized Lipodystrophy (GL)

Lorelei lives with acquired generalized lipodystrophy (GL).

Once Lorelei started Myalept to replace her missing leptin, her triglycerides and blood glucose numbers got better.

From Camerin, Lorelei’s mother:

So many questions

One day, when Lorelei was almost three years old, I noticed lumps on her forehead and her cheeks were rock hard. She was listless, tired, and running a fever. We ended up in the hospital for two weeks and every specialist we saw seemed to have no idea what Lorelei had.

They did blood work and tests, and we found out she also had swollen lymph nodes all over her body and an enlarged liver and spleen. We later learned the fat cells in her body were hardening and she was diagnosed with panniculitis, which we were told was a symptom of something else. Within two months, all the fat in her body was gone and it’s never come back.

We saw many different specialists, each with their own possible diagnosis, but each test was negative and only left us with more questions.

A balancing act

From the time Lorelei was 3 to 5 years old, she practically spent more time in the hospital than at home, being hospitalized a total of 13 times. It took almost a year, tons of blood work, and numerous biopsies to get a definitive diagnosis. But finally, she was diagnosed with acquired GL.

Because of the lack of fat and leptin caused by GL, she developed severe insulin resistance and diabetes, which made a complicated situation more difficult. And without leptin, her blood glucose and triglyceride levels were very high. It also wasn’t until a scary episode of pancreatitis that we discovered her body didn’t process fat correctly. That’s when we, including the doctors, learned just how complicated GL could be.

I came to understand that GL is a disease that causes a loss of fat tissue all over her body and causes a lack of leptin. Lorelei didn’t have enough leptin and we struggled trying to get her triglycerides and blood glucose levels down, and she was hospitalized many times.

Living her life

We saw many more specialists—gastroenterologists, rheumatologists, endocrinologists, a pediatric hepatologist. We learned about treatment options and about Myalept, a leptin replacement therapy which was then in clinical trial. It was explained to us how, together with diet, Myalept may help lower her high blood glucose and high triglycerides.

Before starting Myalept, we were trained by her nurse on how to give her injections. Lorelei had a skin reaction to her first Myalept injection, but she’s been able to take her injections well ever since. And the best part was that we saw results within months. Her triglycerides and blood glucose numbers got better. Of course, this has just been her experience.

Lorelei is an amazing little girl. I think sometimes adults pity themselves so much when they have a disease, yet she just runs around, plays, and lives her life. Of course, she still gets sick sometimes and has a lot going on with shots and medicines every day, but she’s a happy little girl, and I’m a proud momma!

Select Important Safety Information

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 triglyceride levels, or use insulin or sulfonylurea.

Camerin’s daughter, Lorelei, lives with GL and is taking Myalept, and Camerin 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 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 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.