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Safety &
Effectiveness

Actor portrayal. MLD is a recessive inherited disease.
Actor portrayal. MLD is a recessive inherited disease.

Safety & Effectiveness

LENMELDY IS PROVEN SAFE AND EFFECTIVE FOR EARLY ONSET* MLD

AFTER TREATMENT WITH LENMELDY

37 children were evaluated for efficacy in clinical trials.

20 of the children had been diagnosed with PSLI MLD, 7 with PSEJ MLD, and 10 with ESEJ MLD.

CHILDREN WITH PSLI MLD

Survival

14 children treated with LENMELDY were alive and had reached the age of 6 years old, while 10 of the untreated children had passed away before the age of 6.

Severe motor impairment

17 children with PSLI MLD treated with LENMELDY were followed until at least the age of 5 years.

At the age of 5 years, 100% of LENMELDY-treated PSLI children remained free of severe motor impairment (loss of movement and loss of sitting without support). All of the untreated children experienced loss of movement and loss of sitting without support by the age of 5.

12 children treated with LENMELDY who had reached the age of at least 5 years (up to 13 years old) were able to walk independently at their last follow-up.

Lower cognitive impairment

Cognitive impairment can include difficulties with thinking and problem solving.

19 of the 20 children with PSLI MLD remained free of severe cognitive impairment through last follow-up.

Most of the LENMELDY-treated PSLI children maintained normal cognition based on standard performance scores at last follow-up. The untreated children demonstrated severe cognitive impairment early in their disease.

CHILDREN WITH PSEJ MLD

Motor function

In the trial, 7 children with PSEJ MLD were treated with LENMELDY.

At the time of follow-up, 1 child had passed away from a stroke, and 3 children were too young to evaluate since symptoms may not show up until 7 years of age.

3 of the children with PSEJ MLD at last follow-up who were old enough to evaluate had normal gait and were walking without support (follow-up occurred at least 3 years post-treatment). Of the 2 untreated matched siblings, only 1 was able to walk without assistance.

Cognitive function

2 children were evaluated and had normal cognitive function approximately 5 to 7 years after being treated.

CHILDREN WITH ESEJ MLD

10 children with ESEJ MLD were studied

At last follow-up, 1 child could not be evaluated due to the progression of their disease, and 2 children had passed away from MLD. All 7 remaining children were evaluated from 2 to 9 years after LENMELDY treatment.

Of the remaining 7 children in the study, 6 children had normal cognitive function

5 children were able to sit, crawl, or roll by themselves, but were unable to walk without assistance.

*See full indication.

WHAT OTHER MEDICINES WILL MY CHILD NEED?

Other medicines, including chemotherapy, are included as part of treatment with LENMELDY. It is important to talk to your doctor about the risks and benefits of all medicines involved in your child’s treatment.

WHAT YOUR CHILD SHOULD AVOID AFTER RECEIVING LENMELDY, AND INFORMATION ABOUT POSSIBLE OR LIKELY SIDE EFFECTS

Children should not donate blood, organs, tissues, or cells. While receiving chemotherapy to prepare their body for LENMELDY, your child may experience nausea, vomiting, decreased appetite, constipation, abdominal pain, headache, and rash.

Following treatment your child may experience:

  • Low blood counts leading to a risk of bleeding and/or infection. Until blood counts (platelets, white blood cells, red blood cells) return to safe levels, your child may be treated with blood and platelet transfusions and other medicines that prevent bleeding and infection by increasing their blood counts. Most children’s blood counts return to safe levels about 1 month after treatment with LENMELDY. Some children’s blood counts may not recover for >1 year.
  • Life-threatening infections. Children treated with LENMELDY may experience serious or life-threatening infections, including infections of the bloodstream by bacteria or viruses. Most infections occur in the first 1 or 2 months after treatment with LENMELDY but can occur >1 year later. Tell your doctor right away if your child develops fever, chills, or any signs or symptoms of an infection.
  • LENMELDY will not give your child human immunodeficiency virus (HIV) infection. Treatment with LENMELDY may cause a false-positive HIV test result by some commercial tests. If your child needs an HIV test, talk with your doctor about the appropriate test to use.
  • Inflamed and painful mouth (typically occurs during the first 2 months after LENMELDY), nausea, vomiting, decreased appetite, constipation, abdominal pain, diarrhea, headache, and new onset seizures may occur.

Treatment with LENMELDY may cause brain inflammation. Tell your doctor right away if your child develops weakness, decreased muscle tone, loss of mental clarity, vomiting, or swallowing difficulties.

These are not all the possible side effects of LENMELDY. Call your doctor for medical advice about side effects. 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.

IMPORTANT SAFETY INFORMATION

What is LENMELDY?

LENMELDY is a one-time gene therapy developed to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile and early symptomatic early juvenile, referred to as early-onset, metachromatic leukodystrophy (MLD). MLD is caused by a defect in the arylsulfatase A (ARSA) gene, which causes the body to produce reduced or no ARSA enzyme. LENMELDY is made specifically for each child, using the child’s own blood stem cells, and adding functional copies of the ARSA gene to their cells. This may allow you to produce sufficient ARSA enzyme to stop or slow the progression of MLD symptoms.

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