This information is for US healthcare professionals only.
Click “continue” only if you are a US healthcare professional.
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
Controlling how fat is broken down or stored. When fat is not used and stored properly in people with 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.
Myalept comes in a powdered form and needs to be mixed with a liquid before injecting. The liquid used to mix Myalept for newborns is different from the liquid used for children and adults. It is very important to follow your doctor’s instructions for mixing Myalept.
Before starting Myalept, your doctor should show you how to inject Myalept for the ﬁrst time. It is important that your doctor watch your ﬁrst injection of Myalept.
Myalept needs to be taken once a day, at the same time each day. You should ﬁnd a time that works for you before starting treatment.
Myalept should be mixed with sterile water for injection (preservative free) (WFI) and used right away. You should throw away any medication not used during each injection. 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 should be mixed with bacteriostatic water for injection (BWFI) and can be used for more than 1 dose for up to 3 days. Mixed Myalept should be stored in the refrigerator and out of the light. You should throw away any medication not used after 3 days.
Once you start taking Myalept, your doctor may adjust your dose of insulin to help reduce the risk of low blood sugar (hypoglycemia). Make sure to review the complete Instructions for Use before each injection.
Call 1-855-6MYALEPT (1-855-669-2537), Monday through Friday, 8 am to 8 pm ET.
In 2014, Myalept was approved by the FDA to be used along with a doctor-recommended diet to treat complications of not having enough leptin in people with generalized lipodystrophy (GL). It is not known if Myalept is safe and effective when used to treat problems 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.
“After I started taking Myalept, my triglyceride levels started going down.”
“After I started taking Myalept, my triglyceride levels started going down.”
Select Important Safety Information
Before using Myalept, tell your doctor if you have any medical conditions including if you are pregnant or plan to become pregnant as it is unknown if Myalept will harm your unborn body. 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 planning or plan to nurse. You should not nurse while you take Myalept.
Dwanna lives with GL, is taking Myalept, and has agreed to share her 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.
When I was born, my mother noticed my skin wasn’t smooth like other babies and was leathery to the touch. I didn’t have any baby fat anywhere on my body, including my face. She knew right away that there was something different with me. She immediately had me checked by the doctor, but no matter what, my diagnosis was inconclusive.
Growing up, I was much taller than my classmates, muscular, and I had very large hands and feet. It was because of my GL but we didn’t know it. Having this strange condition, my mother took me back and forth to the hospital for numerous tests. I dreaded those appointments. But apart from dealing with the bullies in grade school who made fun of my appearance calling me “skinny” and “muscle-bound,” I had a pretty normal life. I was very strong and athletic. I became very good at sports and enjoyed playing just about everything. When I broke my elbow, I had to stop participating in sports.
Later, I was diagnosed with diabetes, which I now know was from the lack of leptin in my system.
I was also always hungry and constantly eating, my blood sugar and triglycerides were often very high, and even though I was taking insulin, my diabetes was extremely insulin-resistant. Finally, when a doctor who was treating me realized I’d been seen at that same hospital years earlier, he checked my medical records and saw “lipodystrophy” written on my chart. He had me tested for it, and the results came back with a diagnosis of congenital generalized lipodystrophy. After 16 years, I finally had a name for it.
In 2000, my doctor approached my mother about having me participate in a clinical trial for a leptin replacement therapy, later called Myalept, and we all agreed.
I’d been taking high doses of insulin for my diabetes because I was so insulin-resistant, but shortly after starting Myalept, my blood sugar was dropping so low I’d prop a chair in front of my refrigerator at night so I could eat immediately when I felt hypoglycemia coming on. Apparently, the leptin replacement was working to lower my blood glucose, so, because I was still taking insulin, I had to watch out for hypoglycemia. My doctor then told me I needed to reduce my insulin to avoid hypoglycemia.
We quickly saw drops in my glucose and triglyceride numbers, and my doctors were very happy with my progress. My cholesterol levels came down, my triglycerides came down, and my diabetes was well controlled. Of course, everyone is different, and their experiences with treatment may be different than mine.
When I look back on my life, I feel fortunate that Myalept is available for people like me who are living with GL.
GL is a Disease of Leptin Deﬁciency.
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.
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:
You should not take Myalept if you:
Before using Myalept, tell your doctor if you have any medical conditions including if you:
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.