Can Myalept (metreleptin) be used for people with diabetes or high cholesterol?

No, Myalept (metreleptin) is only for people who are diagnosed with GL. Talk to your provider about available [treatment options for diabetes](https://www.goodrx.com/conditions/diabetes-type-2#medications) or [high cholesterol](https://www.goodrx.com/conditions/high-cholesterol#medications). They can discuss which option is right for you.

Myalept

Q: What is generalized lipodystrophy?

[Generalized lipodystrophy (GL)](https://myalept.com/gl-leptin/) is a rare genetic condition that causes people to have little to no body fat. This results in low [leptin](https://www.yourhormones.info/hormones/leptin/) levels. Leptin is a hormone released by the fat cells in your body. It regulates your [metabolism](https://medlineplus.gov/ency/article/002257.htm) (how your body uses calories for energy) and helps control your body weight. [Complications](https://www.atherosclerosis-journal.com/article/S0021-9150(18)30782-2/fulltext) of GL include high triglyceride (a type of fat) levels, high blood sugar levels (or type 2 diabetes), and [fatty liver](https://medlineplus.gov/genetics/condition/non-alcoholic-fatty-liver-disease/). Myalept (metreleptin) helps with GL, because it replaces the leptin your body is missing.

Q: Can I suddenly stop taking Myalept (metreleptin)?

No, don't stop taking Myalept (metreleptin) without notifying your provider first. This is especially important if you've had [pancreatitis](https://www.goodrx.com/conditions/pancreatitis) (inflammation of the pancreas) or have severely high triglyceride levels. Stopping the medication suddenly can raise your risk of developing these problems. Let your provider know if you want to stop Myalept (metreleptin) so your dose can be slowly and safely lowered.

Q: Where should I inject Myalept (metreleptin)?

You can give the Myalept (metreleptin) injection under the skin in a body area that has the most fat. These locations include your thigh, back of your upper arm, and stomach. Never inject the medication into your muscle or vein. To lower the risk of bruising and irritation, rotate between different injection sites each time you use the medication so that the same site isn't used repeatedly.

Q: Can I use Myalept (metreleptin) if I'm pregnant?

Myalept (metreleptin) hasn't been well studied in human pregnancy, so it's not known whether this medication is safe or works well in pregnant females. Some animal studies that looked at Myalept (metreleptin) use during pregnancy showed that babies weren't harmed from the medication. If you're pregnant or thinking of becoming pregnant, talk with your provider about the risks and benefits of using Myalept (metreleptin) during pregnancy. If you're pregnant and are prescribed Myalept (metreleptin) or become pregnant during treatment, it's recommended that you enroll in a pregnancy exposure registry by calling 1-855-669-2537. This registry can monitor your and your unborn baby's health.

metreleptin

Used for Lipodystrophy

Myalept (metreleptin) is the only medication approved for use in people with a rare genetic condition called generalized lipodystrophy (GL). This medication is added onto a dietary plan that your provider recommends you to follow. It's given as an injection under the skin once per day, but is available as brand only, so it can be expensive.

Reviewed by:

Robert Chad Hakim, PharmD, BCCCP

Allison Gourley, PharmD

Last reviewed on August 11, 2022

What is Myalept (metreleptin)?

What is Myalept (metreleptin) used for?

Congenital generalized lipodystrophy (CGL)
Acquired generalized lipodystrophy (AGL)

How Myalept (metreleptin) works

Myalept (metreleptin) is a leptin analog. Leptin is a hormone made in your body that helps regulate (maintain) body weight. It helps use and store fat properly, which affects the level of triglycerides (a type of fat) in your body. Leptin also boosts your body's ability to use blood sugar as energy.

By acting like leptin, Myalept (metreleptin) helps keep the levels of triglycerides and blood sugar low in people with GL who have little to no leptin in their body.

Drug facts

Common Brands	Myalept
Drug Class	Leptin analog
Controlled Substance Classification	Not a controlled medication
Generic Status	No lower-cost generic available
Availability	Prescription only

Common Brands

Myalept

Drug Class

Leptin analog

Controlled Substance Classification

Not a controlled medication

Generic Status

No lower-cost generic available

Availability

Prescription only

Side effects of Myalept (metreleptin)

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Headache (13%)
Low blood sugar (13%)
Weight loss (13%)
Stomach pain (10%)

Less Common Side Effects

Back or joint pain
Dizziness
Fatigue
"Pins and needles" feeling
Nausea
Diarrhea
Fever
Upper respiratory tract infection
Ear infection
Anemia
Ovarian cyst

Myalept (metreleptin) serious side effects

Contact your healthcare provider immediately if you experience any of the following.

Low blood sugar when used with certain medications: feeling faint, shaking, sweating, dizziness, fast heartbeat, nervousness, anxious, confused, irritable, feeling very hungry
Allergic reactions: hives, rash, blisters, skin reddening, swelling of the lips or tongue, difficulty breathing

Source: DailyMed

The following Myalept (metreleptin) side effects have also been reported

Side effects that you should report to your care team as soon as possible:

Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat

Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):

Dizziness
Fatigue
Headache
Joint pain
Stomach pain

Pros and cons of Myalept (metreleptin)

Pros

Works well as an add-on treatment to diet for GL
Injected once per day
Can be given at home after being properly trained
Available only through a Risk Evaluation and Mitigation Strategies (REMS) program to make sure it's as safe for you as possible

Cons

Might be hard for some people to administer correctly
Needs to be injected under the skin
Can lower blood sugar even more if using with certain diabetes medications (e.g., insulins, glipizide (Glucotrol))
Brand name only, which can be expensive

Pharmacist tips for Myalept (metreleptin)

Make sure your provider or pharmacist teaches you how to prepare and give Myalept (metreleptin) injections. Learning to use Myalept (metreleptin) correctly will help avoid accidental injuries or medication waste. Carefully read the instructions on how to use the medication. Contact your provider or pharmacist if you have any questions.
If your child weighs 88 pounds or less, your provider or pharmacist might give you an insulin syringe to use to draw up your child's Myalept (metreleptin) dose. This is because regular syringes might be too big to use, which can raise the risk of giving your child the incorrect dose. If you're using insulin syringes, ask your provider or pharmacist to teach you to draw the correct dose of Myalept (metreleptin).
Store Myalept (metreleptin) in the refrigerator, and keep the vials in the original carton away from light. Don't freeze them.
When it's time for a dose, take the medication out of the refrigerator, and allow it to reach room temperature (takes about 10 minutes). Don't try to warm up the medication any other way (e.g., microwave, under hot water), because you might damage it.
If you mix the Myalept (metreleptin) vial with sterile water for injection, you can only use the prepared medication for one dose. Throw away any leftover medication, even if there's still some remaining in the vial. The mixture doesn't contain any preservatives to keep germs and bacteria out of the medication once you open it.
If you mix the Myalept (metreleptin) vial with bacteriostatic water for injection (for older children and adults only), you can use the prepared medication for up to 3 days when stored in the refrigerator after each dose and protected from light. After the third day, throw out the vial, even if there's still some medication remaining.
Make sure to shake the vial gently after preparing the medication. Don't shake it too hard, because this might damage the medication.
Throw away used syringes and needles in a sharps container or something made of heavy-duty plastic (e.g., empty milk carton, laundry detergent bottle). This helps lower the risk of needle-stick injuries. Don't throw them away in your trash can. For more information on how to throw away your sharps container safely and guidance for your specific state, visit the FDA's website.

Common questions about Myalept

Generalized lipodystrophy (GL) is a rare genetic condition that causes people to have little to no body fat. This results in low leptin levels. Leptin is a hormone released by the fat cells in your body. It regulates your metabolism (how your body uses calories for energy) and helps control your body weight. Complications of GL include high triglyceride (a type of fat) levels, high blood sugar levels (or type 2 diabetes), and fatty liver. Myalept helps with GL, because it replaces the leptin your body is missing.

No, don't stop taking Myalept without notifying your provider first. This is especially important if you've had pancreatitis (inflammation of the pancreas) or have severely high triglyceride levels. Stopping the medication suddenly can raise your risk of developing these problems. Let your provider know if you want to stop Myalept so your dose can be slowly and safely lowered.

No, Myalept is only for people who are diagnosed with GL. Talk to your provider about available treatment options for diabetes or high cholesterol. They can discuss which option is right for you.

You can give the Myalept injection under the skin in a body area that has the most fat. These locations include your thigh, back of your upper arm, and stomach. Never inject the medication into your muscle or vein. To lower the risk of bruising and irritation, rotate between different injection sites each time you use the medication so that the same site isn't used repeatedly.

Myalept hasn't been well studied in human pregnancy, so it's not known whether this medication is safe or works well in pregnant females. Some animal studies that looked at Myalept use during pregnancy showed that babies weren't harmed from the medication. If you're pregnant or thinking of becoming pregnant, talk with your provider about the risks and benefits of using Myalept during pregnancy. If you're pregnant and are prescribed Myalept or become pregnant during treatment, it's recommended that you enroll in a pregnancy exposure registry by calling 1-855-669-2537. This registry can monitor your and your unborn baby's health.

Risks and warnings for Myalept (metreleptin)

Myalept (metreleptin) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

Risk of developing neutralizing antibodies

Warning Icon It's possible for Myalept (metreleptin) to cause your body to make antibodies against leptin or the medication itself and inactivate them. Though not completely clear, this could mean that the medication could start to not work as well, your leptin levels could drop, and certain signs or symptoms of GL could appear. This could include severe infections, diabetes (e.g., high blood sugar, excessive thirst or urination), weight gain, or high triglyceride levels. There are currently ongoing studies that are trying to better understand the clinical impact of this problem.

Potential risk for lymphoma

Risk factors: Acquired GL | Low white blood cell count | Bone marrow problems | Lymph node problems (e.g., lymphoma, swollen lymph nodes)

Warning Icon Although rare, there were reports of lymphoma (a type of immune system cancer) in people taking Myalept (metreleptin) during clinical studies. This only happened in three people using the medication for acquired GL who also had a weakened immune system and bone marrow problems. While there might be a potential risk for lymphoma when treated with Myalept (metreleptin), it's not clear if the medication is linked to this cancer. If you have acquired GL, low blood cell counts, bone marrow problems, or lymph node problems, or if you have any concerns, talk to your provider about this potential risk.

Myalept REMS program

There are potential risks for lymphoma (a type of immune system cancer) and for the development of antibodies that attack leptin in your body. Because of these risks, Myalept (metreleptin) is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myalept REMS program. All providers who prescribe Myalept (metreleptin) and pharmacies that dispense the medication must register with the REMS program. Under this program, your provider must discuss the potential risks of Myalept (metreleptin) treatment with you. This program is to make sure the medication is distributed and taken under the safest conditions possible for you.

Low blood sugar when taken with certain medications

Risk factors: Taking medications that lower your blood sugar (e.g., insulin, sulfonylureas)

If you're taking certain other medications that can lower blood sugar (e.g., insulins, sulfonylureas), using Myalept (metreleptin) with these medications can drop your blood sugar even lower to dangerous levels. Tell your provider if you're taking any of these medications, since they might need to adjust your doses to lower your risk. It's important to know how to recognize symptoms of low blood sugar and how to treat it. Always keep emergency glucose tablets or hard candy on you in case your blood sugar drops too low. If you feel faint or experience shaking or sweating, check your blood sugar and treat episodes of low blood sugar right away. If your symptoms don't improve, call 911.

Severe allergic reactions

Severe allergic reactions to Myalept (metreleptin) are possible, including life-threatening reactions like facial swelling, shock, and anaphylaxis (e.g., closing of the throat). If you notice difficulty breathing, a choking sensation, dizziness, nausea, or swelling around your eyes, lips, or tongue after using Myalept (metreleptin), get medical attention right away.

Serious benzyl alcohol toxicity in young children

To prepare Myalept (metreleptin), the medication can be mixed with sterile water for injection or bacteriostatic water for injection. Bacteriostatic water for injection contains benzyl alcohol, which is a preservative that has caused serious side effects, including death, in children (particularly in newborns and premature babies). If your young child needs to use Myalept (metreleptin), mix the medication with only preservative-free sterile water for injection.

Myalept (metreleptin) dosage

Typical dosage for Myalept (metreleptin)

Myalept (metreleptin) is given as an injection under the skin. Your provider will adjust the dose based on how you're responding to the medication.

People weighing 88 lbs (40 kg) or less: The typical starting dose is 0.06 mg/kg of body weight once per day. The maximum dose is 0.13 mg/kg once per day.
Males weighing more than 88 lbs: The typical starting dose is 2.5 mg once per day. The maximum dose is 10 mg once per day.
Females weighing more than 88 lbs: The typical starting dose is 5 mg once per day. The maximum dose is 10 mg once per day.

Myalept (metreleptin) contraindications

Contraindications are specific health conditions or situations in which a person should not take a medication due to safety concerns. If you have any of the following conditions or if any of the following apply to you, let your healthcare provider know because Myalept (metreleptin) will not be safe for you to take.