Press Release
☷FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy
Food and Drug Administration ( By Press Release office)
Mar 20,2024
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Hey everyone , just wanted to give you a heads up that the FDA has approved Lenmeldy ( atidarsagene autotemcel ) for kids with metachromatic leukodystrophy . It ' s the first gene therapy approved for this rare genetic disease that affects the brain and nervous system . Lenmeldy is a one - time infusion made from the patient ' s own stem cells , which have been modified to include a functional ARSA gene . This could potentially stop the progression of MLD and improve outcomes for patients . The approval is a big step forward in providing effective treatments for rare diseases like MLD . The FDA reviewed data from 37 children who received Lenmeldy in clinical trials to assess its safety and effectiveness . Kids who got treated with Lenmeldy were compared to those who didn ' t receive any treatment . The main goal was to see how long they could maintain their ability to move without help or sit up without support before experiencing a decline or passing away . In children with MLD , getting treated with Lenmeldy significantly lowered the chances of losing motor function or dying compared to those who weren ' t treated . All kids with early signs of MLD who received Lenmeldy were still alive at 6 years old , while only 58% of untreated kids made it to the same age . By 5 years old , 71% of treated kids could walk on their own . 85% of treated kids had normal language and performance IQ scores , which wasn ' t seen in untreated kids . Kids with different types of MLD also showed some improvement in their motor and cognitive functions after treatment . The most common side effects of Lenmeldy include fever , low white blood cell count , mouth sores , infections , rash , and liver enlargement . Patients should be closely monitored for any changes in blood counts and brain swelling after receiving Lenmeldy . While there is a small risk of developing blood cancer with this treatment , no cases have been reported in patients who received Lenmeldy . Patients should be monitored for potential blood cancers for at least 15 years after treatment . Lenmeldy was granted approval by the FDA to Orchard Therapeutics after receiving several designations .
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