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.
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.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):
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
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
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.
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.
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.
Risk factors: Acquired GL | Low white blood cell count | Bone marrow problems | Lymph node problems (e.g., lymphoma, swollen lymph nodes)
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.
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.
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 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.
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) 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.
Identify as obese, and body weight isn't caused by GL
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