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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 extended life expectancy in children with PSLI and reduced the impact of early onset* MLD

LENMELDY is a transformative treatment for children with early onset* MLD in the US

Results from studies including 37 children treated with LENMELDY

20 infants with PSLI MLD, aged under 30 months without symptoms

LENMELDY significantly extended overall survival rates and reduced or prevented impairment

Overall survival of the children who took part in the study from birth up to 6 years old:

LENMELDY Group
All still alive
Untreated Group
10 passed away

Move and sit without support (severe motor impairment):

LENMELDY Group
All kept their ability to move or sit without support
Untreated Group
None kept their ability to move and sit without support by the age of 5

Walk independently:

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

Maintain thinking and problem-solving abilities:

LENMELDY Group
Only 1 child demonstrated difficulties with thinking and problem-solving (severe cognitive impairment)
Untreated Group
All children demonstrated difficulties with thinking and problem-solving (severe cognitive impairment) early in their disease
7 young children with PSEJ MLD, aged 30 months to 7 years without symptoms

LENMELDY helped children maintain their ability to think and walk by themselves:

At the time of the last follow-up, 3 of the 7 children treated with LENMELDY were eligible for walking assessments and 2 for cognitive functioning assessments:

All children (3) kept their ability to walk on their own
All children (2) had normal levels of thinking and problem-solving approximately 5 to 7 years after being treated
10 young children with ESEJ MLD, aged 30 months to 7 years with early-stage symptoms

LENMELDY helped children keep their mental and physical abilities:

At the time of the last follow-up, 7 of the 10 children treated with LENMELDY were eligible for assessment from 2 to 9 years after LENMELDY treatment:

All children (7) maintained normal levels of thinking and problem-solving
5 of the 7 children were able to sit, crawl, or roll by themselves (but were unable to walk without help)

Hear one father talk about his daughter’s experience with LENMELDY

Watch Video
* See full indication.
  ESEJ, early symptomatic early juvenile; MLD, metachromatic leukodystrophy; PSEJ, pre-symptomatic early juvenile; PSLI, pre-symptomatic late infantile.

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

Patients 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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