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Overview: Lenmeldy is a gene therapy that is given as a one-time infusion into a blood vessel. Lenmeldy is used for certain people with a rare, inherited problem called metachromatic leukodystrophy (MLD). Lenmeldy is made from the person’s own stem cells. Common side effects may include infections, mouth sores, and rash. Tell your health care provider right away if you get a fever or unusual bleeding or bruising. Get emergency help if you have chest pain or trouble breathing or seeing.

Uses

What is Lenmeldy used for?

Lenmeldy (atidarsagene autotemcel) is commonly used to treat certain types of a condition called metachromatic leukodystrophy (MLD). This rare, inherited condition causes breakdown of the covering around nerves, called myelin.

Lenmeldy may also be used for other conditions as determined by your health care provider.

How does Lenmeldy work (mechanism of action)?

People with metachromatic leukodystrophy (MLD) do not have enough of a functioning protein called arylsulfatase A (ARSA). As a result, a build-up of harmful substances that damages nerves occurs. 

Lenmeldy is a gene therapy that provides the needed instruction to make ARSA. Lenmeldy is made using the person’s own stem cells that are collected from the blood. These cells are changed to include healthy, working copies of the ARSA gene. The changed cells are then infused back into the body, which helps the body make ARSA. Enough of this protein can prevent the build-up of harmful substances that causes nerve problems from loss of myelin. Lenmeldy may help stop or slow the progression of MLD.

How is Lenmeldy supplied (dosage forms)?

Lenmeldy is available in an injectable form that is given by a health care provider.

How should I store Lenmeldy?

Lenmeldy is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Lenmeldy?

The most common side effects of Lenmeldy are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Infection or other problems (such as swelling or redness) where the medicine is infused
  • Infections (see below)
  • Fever
  • Mouth sores
  • Liver problems
  • Runny nose or stuffy nose
  • Rash

There may be other side effects of Lenmeldy that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Lenmeldy?

While less common, the most serious side effects of Lenmeldy are described below, along with what to do if they happen.

Severe Allergic Reactions. Lenmeldy may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Infections. You may have a higher risk of infections, including serious infections that may be life-threatening. This risk may last for up to a year after you get Lenmeldy. Take any medicine given to you to help prevent an infection. Call your health care provider right away if you have an infection or any of the following symptoms.

  • Burning or stinging when you pee 
  • Coughing, or coughing up blood
  • Fever, sweats, chills, sore throat, or muscle aches
  • Shortness of breath
  • Diarrhea, nausea, or stomach pain
  • Weight loss
  • Tiredness
  • Swollen glands

Liver Problem. Some people who have received Lenmeldy have had a liver problem called hepatic veno-occlusive disease. This problem can be life-threatening and happens when small blood vessels in the liver get blocked. Keep all appointments for blood work to check the health of your liver. Call your health care provider right away if you have any of the following symptoms.

  • Stomach or belly pain or swelling
  • Sudden weight gain
  • Nausea or vomiting
  • Weakness or unusual tiredness
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Cancer. Lenmeldy may increase the risk of a new cancer to develop or cause a previously treated cancer to recur. Your health care provider will likely watch you over your lifetime for possible cancer development. Keep all appointments to have your blood checked. Tell your health care provider about any new symptoms that you may get.

Blood Clots. Lenmeldy may cause a blood clot in your arteries or veins. Get emergency help if you have any of the following signs of blood clots. 

  • Chest pain or pressure
  • Fast or abnormal heartbeat
  • Trouble breathing
  • Pain in your back, jaw, throat, or arm
  • Sweating, upset stomach, vomiting, or dizziness
  • Sudden weakness on one side of your body
  • Sudden and severe headache
  • Slurred speech or trouble understanding speech
  • Pain, swelling, warmth, discoloration, or tenderness in your arm or leg
  • Vision loss
  • Bulging eyes 
  • Double vision

Brain Inflammation. Lenmeldy may increase your risk of encephalitis. Call your health care provider right away if you have any of the following symptoms.

  • Stiff neck
  • Balance problems 
  • Memory problems or confusion
  • Muscle weakness
  • Behavior problems
  • Trouble swallowing
  • Vomiting

Warnings & Precautions

Who should not use Lenmeldy?

Allergies to Ingredients. People who are allergic to any of the following should not use Lenmeldy.

  • Atidarsagene autotemcel
  • Lenmeldy 
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Lenmeldy.

What should I know about Lenmeldy before using it?

Lenmeldy is usually given by a health care provider in a hospital or clinic. It is given as a one-time infusion into a vein (IV).

It is very important to keep all appointments for bloodwork and with your health care provider before and after receiving Lenmeldy. These appointments help your health care provider make sure the treatment is safe and working well for you. Tell your health care provider about any new or worsening symptoms, especially if you have easy bruising or bleeding, bleeding that does not stop, or nosebleeds.

Tell all of your health care providers that you have received Lenmeldy. Lenmeldy may affect the results of certain lab tests. Also, if you need any blood products like a blood transfusion after receiving Lenmeldy, the blood may need to be treated with a special process first to make it safer for you.

You should not donate blood, organs, cells, or tissues at any time after receiving Lenmeldy. 

If you can get your partner pregnant, you should use an effective method of birth control before you take the steps needed to receive Lenmeldy and for at least 6 months afterwards. See the Pregnancy section for more information.

Before receiving Lenmeldy, talk to your health care provider if you or your child may want to become pregnant or have a partner become pregnant in the future. 

There is a small risk that the changed stem cells may not grow properly in your body. Your health care provider will likely keep a back-up supply of your cells before you receive Lenmeldy in case they are needed later. If you have any questions, ask your health care provider.

What should I tell my health care provider before using Lenmeldy?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Lenmeldy is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

Vaccines. Talk with your health care provider about any vaccines that you may need. You should not receive vaccines for certain lengths of time both before and after receiving Lenmeldy. Tell your health care provider about any vaccines you have recently received or have scheduled.

Pregnancy. People who can become pregnant are not usually prescribed Lenmeldy. It is not known if or how Lenmeldy could affect pregnancy or harm a fetus. If you can get pregnant, you should have a test to make sure you are not pregnant before you take the steps needed to receive Lenmeldy. You should also use effective birth control before you take the steps needed to receive Lenmeldy. Tell your health care provider right away if you are pregnant, think you may be pregnant, or plan to become pregnant.

Breastfeeding. People who can breastfeed are not usually prescribed Lenmeldy. It is not known if Lenmeldy passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should receive Lenmeldy while breastfeeding.

How can I get more information about Lenmeldy?

For more information about Lenmeldy, you can visit the drugmaker’s website at www.lenmeldy.com.

Interactions

Does Lenmeldy interact with foods or drinks?

There are no known interactions between Lenmeldy and foods or drinks.

It is unknown if drinking alcohol will affect Lenmeldy.

Does Lenmeldy interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are taking any medicines for either treatment or prevention of HIV before receiving Lenmeldy.

This may not be a complete list of medicines that can interact with Lenmeldy. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Lenmeldy?

If you or someone else has used too much Lenmeldy, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Lenmeldy?

Lenmeldy is usually given by a health care provider in a hospital or clinic. If you miss your appointment to receive Lenmeldy, call to make another appointment as soon as possible.