On March 19, the FDA granted approval to Orchard Therapeutics’ gene-edited cell therapy atidarsagene autotemcel (now branded as Lenmeldy in the U.S.) for treating children with metachromatic leukodystrophy (MLD). It is the first gene therapy for children suffering from this rare hereditary disorder to be approved in the United States. The disease is estimated to affect one in every 40,000 individuals in the United States, according to the FDA.

Orchard, acquired by Japanese pharmaceutical firm Kyowa Kirin last year for $477.6 million, specified that the treatment pricing and availability details would be revealed soon.

The approval decision was based on data from 37 of the aforementioned 39 patients. The results of the two open-label clinical trials unveiled that the therapy significantly reduced the risk of severe motor impairment or death compared with untreated children. The FDA added that all children in this group with presymptomatic (PS) late infantile (LI) MLD were alive at six years of age; on the other hand, just 58% of children with PS LI MLD in the natural history group were still living at six years of age. In addition, 71% of children treated with arsa-cel could walk without assistance at five years of age. Furthermore, 85% of children treated with arsa-cel had normal language and performance IQ scores, a phenomenon not reported in untreated patients. The agency also noted that slowing of motor and cognitive disease was observed in patients with presymptomatic early juvenile (PS EJ) and early symptomatic early juvenile (ES EJ) MLD treated with arsa-cel.

The FDA also notes that until engraftment has occurred, patients who have received arsa-cel should be monitored for neutrophil counts and risk of delayed platelet engraftment. Furthermore, although no cases of blood cancers have been observed in patients who received arsa-cel, the FDA lists it as a potential risk and advises lifelong monitoring for such cancers. MLD, which affects the brain and nervous system, is characterized by the buildup of certain fatty substances in the cells, leading to loss of motor and cognitive function and early death.

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