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FDA approves Orchard’s Lenmeldy gene therapy for MLD

2024-03-19

基因疗法上市批准临床研究

In clinical studies, 85% of children treated with Lenmeldy maintained normal language and performance IQ scores. Credit: BlueBoeing / Shutterstock.com.

The US Food and Drug Administration (FDA) has granLenmeldyoval to Orchard Therapeutics‘ Lenmeldy (atidarsagene autotemcel) gene therapy for use in children with specific forms of metachromatic leukodystrophy (MLD).

This is the first FDA approval for a gene therapy addressing pre-symptomatic late infantile, early juvenile or early symptomatic early juvenile MLD.

LenmeldFood and Drug Administration (FDA)fusion, utilising the patOrchard TherapeuticsodLenmeldymaatidarsagene autotemcelSCs) to halt disease progression.metachromatic leukodystrophy (MLD)

The gene therapy iFDAlves collecting HSCs from the patient and modifying them to include functional copies of the ARSA gene.MLD

Lenmeldyfied cells are re-introduced into the patient’s bone marrow where they engraft and produce immune cells that can break down sulfatides, the substances that accumulate harmfully in MLD.

See Also:ARSA

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Everest receives Singapore HSA approval for NEFEGAN to treat IgAN

Johnson & Johnsone previously received high-dose chemotFDAapy to prepare the bone marrow for the modified cells.

The efficacy and safety of the therapy were evaluated based on data from 37 children who participated in two single-arm, open-label clinical trials and an expanded access programme.

Severe motor impairment-free survival, definNEFEGANhe time frIgANirth to the first appearance of locomotion loss, loss of sitting without support, or mortality, was the primary measure of efficacy.

All children in the treated group with pre-symptomatic late infantile MLD were alive at six years old, in contrast to only 58% in the natural history group.

71% of treated children could walk unassisted at five years of age.

85% of treated children maintained normal language and performance IQ scores, a result not observed in untreated children.

Those wmotor impairmenttic early juvenile and early symptomatic early juvenile MLD exhibited a deceleration in motor and cognitive disease progression.

FDA Center for Biologics Evaluation and Research director Peter Marks stated: “This is the first FDA-approved treatment option for children with this rare genetic disease.

“We remain committed to advancing scientific and regulatory principles that enable the efficient development and review of safe, effective and innovative products that have the potential to change patients’ lives.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards oMLDournalistic integrity. Topic sponsors are not involved in the creation of editorial content.

FDA Center for Biologics Evaluation and Researcham to pipelinerare genetic disease

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