Home Medizin FDA genehmigt Gentherapie für metachromatische Leukodystrophie

FDA genehmigt Gentherapie für metachromatische Leukodystrophie

von NFI Redaktion

The Food and Drug Administration (FDA) in the United States has approved the first gene therapy for metachromatic leukodystrophy (MLD), a rare and debilitating genetic disease that affects the brain and nervous system.

Atidarsagene autotemcel (Lenmeldy) received priority review and was designated as an „Orphan Drug,“ „Rare Pediatric Disease,“ and „Regenerative Medicine Advanced Therapy.“

The approved indication is for pre-symptomatic late-infantile, pre-symptomatic early-juvenile, or early-symptomatic early-juvenile MLD.

„MLD is a devastating disease that profoundly impacts the quality of life for patients and their families. This approval marks an important advancement in the development and availability of effective treatments, including gene therapies, for rare diseases,“ said Nicole Verdun, MD, Director of the Office of Therapeutics in the Center for Biologics Evaluation and Research, said in a statement.

MLD affects an estimated 1 in 40,000 individuals in the United States. There is no cure. Treatment typically focuses on supportive care and symptom management.

MLD is caused by a deficiency of arylsulfatase A (ARSA), which leads to an accumulation of sulfatides in cells, resulting in damage to the central and peripheral nervous system, leading to loss of motor and cognitive functions and early death.

Lenmeldy is a single, personalized dose infusion made from the patient’s hematopoietic stem cells genetically modified to contain functional copies of the ARSA gene.

The safety and efficacy of Lenmeldy were evaluated based on data from 37 children with MLD who received Lenmeldy in two single-arm, open-label clinical studies and through an expanded access program.

In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death (primary endpoint) compared to untreated children (natural history group).

All children with pre-symptomatic late-infantile MLD treated with Lenmeldy were still alive at 6 years of age, compared to only 58% of children in the natural history group.

By the age of 5, 71% of treated children were able to walk independently and 85% had normal speech and performance IQ scores, which were not reported in untreated children.

In addition, children with pre-symptomatic early-juvenile and early-symptomatic early-juvenile MLD showed a slowdown in motor and cognitive decline.

The most common side effects of Lenmeldy are fever and low white blood cell count, mouth sores, respiratory infections, rash, medical device-related infections, viral infections, fever, gastrointestinal infections, and enlarged liver.

„After infusion with Lenmeldy, patients should be monitored for neutrophil count and the risk of delayed platelet engraftment until engraftment is achieved,“ the FDA said.

Treatment with Lenmeldy may be associated with blood clot formation or encephalitis and carries the risk of blood cancer. Patients receiving Lenmeldy should be continuously monitored for hematologic malignancies; this monitoring should include a complete blood count with yearly differential blood count and, if necessary, integrase site analysis for at least 15 years after treatment, according to the FDA.

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