FDA Approves First Gene Therapy Lenmeldy for Rare Brain Disease in Children

• FDA approved Lenmeldy, the first gene therapy for children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system

• Lenmeldy is made from the patient’s own modified blood stem cells to supply the body with cells producing the missing ARSA enzyme

• In clinical trials, Lenmeldy significantly reduced risk of severe motor impairment or death compared to untreated children

• Common side effects include fever, low white blood cell count, infections, etc.

• Patients receiving Lenmeldy should have lifelong monitoring for potential blood cancers and other risks