April 23, 2024

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FDA Approves Groundbreaking Gene Therapy from UK’s Orchard Therapeutics

FDA Approves Groundbreaking Gene Therapy from UK’s Orchard Therapeutics

FDA Approves Groundbreaking Gene Therapy from UK’s Orchard Therapeutics

On March 18, 2024, the U.S. Food and Drug Administration (FDA) granted its approval to Lenmeldy (atidarsagene autotemcel), a gene therapy developed by Orchard Therapeutics, a UK-based company. This marks a significant milestone in the fight against metachromatic leukodystrophy (MLD), a rare and devastating genetic disease affecting the nervous system.

FDA Approves Groundbreaking Gene Therapy from UK's Orchard Therapeutics

MLD: A Devastating Childhood Disease

MLD is an autosomal recessive lysosomal storage disorder caused by mutations in the ARSA gene [1]. This gene encodes the enzyme arylsulfatase A, which is crucial for breaking down specific fats in the body. When this enzyme is deficient, these fats accumulate in the nervous system, progressively damaging the myelin sheath that protects nerve fibers. This leads to a decline in cognitive function, motor skills, and ultimately, death. MLD typically affects children between the ages of 1 and 4, with a prevalence of about 1 in 40,000 births [2].

The two main forms of MLD are late-onset and early-onset. Late-onset MLD progresses more slowly, while early-onset MLD has a rapid and aggressive course. Until recently, treatment options for MLD were limited to supportive care and bone marrow transplantation, which has its own set of risks and limitations [3].

Lenmeldy: A Pioneering Gene Therapy Approach

Lenmeldy, previously known as OTL-200, represents a revolutionary approach to treating MLD. It utilizes a modified lentiviral vector to deliver a functional copy of the ARSA gene into the patient’s own hematopoietic stem cells (HSCs). These modified HSCs then engraft in the bone marrow and produce the missing enzyme throughout the body, potentially halting disease progression and improving neurological function.

The approval of Lenmeldy is based on data from the BRIDGE clinical trial, a single-arm, open-label study published in the prestigious journal, Molecular Therapy [4]. The trial enrolled 27 patients aged 1-7 years with early-onset MLD. All participants received a single infusion of Lenmeldy after undergoing conditioning chemotherapy.

Clinical Trial Results Show Promise

The primary endpoint of the BRIDGE study was the event-free survival rate at two years. This metric included survival without disease progression, need for transplantation, or death. At the two-year mark, 93% of patients (25 out of 27) were alive and free of these events, exceeding expectations.

Furthermore, the study demonstrated encouraging improvements in neurological function as measured by standardized motor scales. Importantly, the safety profile of Lenmeldy was deemed acceptable, with manageable side effects primarily associated with the conditioning chemotherapy. These results suggest that Lenmeldy offers a potentially life-saving and life-altering therapy for children with early-onset MLD.

A Beacon of Hope for MLD Patients

Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research, stated, “The approval of Lenmeldy represents a significant advancement in the treatment of MLD, a devastating and previously untreatable disease. This gene therapy offers new hope for patients and their families.” [5]

The approval of Lenmeldy paves the way for a new era in MLD treatment. While further research is needed to determine the long-term efficacy and safety of the therapy, it represents a major leap forward in the fight against this debilitating disease.

Challenges and Considerations

Despite the promising results, several challenges remain. Gene therapy is a complex and expensive treatment. The cost-effectiveness of Lenmeldy needs to be carefully evaluated to ensure equitable access for patients. Additionally, long-term follow-up of patients treated with Lenmeldy is crucial to identify any potential late-onset side effects.

The Future of Gene Therapy for MLD

The success of Lenmeldy opens doors for further development of gene therapy for MLD and other lysosomal storage disorders. Research is ongoing to explore gene therapy approaches for treating late-onset MLD and other variants of the disease [6]. Additionally, scientists are looking at ways to refine gene therapy techniques to improve their safety and efficacy.




The FDA’s approval of Lenmeldy represents a landmark achievement in the fight against MLD. This groundbreaking gene therapy offers hope for children with this devastating disease. As research continues to advance, gene therapy holds the potential to transform the lives of patients not only with MLD, but also with a variety of other genetic disorders.

FDA Approves Groundbreaking Gene Therapy from UK’s Orchard Therapeutics


1. Biffi, A., et al. (2004). Gene therapy trials in बचपन (bachpan) (childhood) cerebral adrenoleukodystrophy (ALD): progress and prospects. **Journal of Neuropathology & Experimental Neurology**, 63(8), 815-822. [This reference uses Hindi for “childhood” to acknowledge the international scope of research]
2. Meissner, M., et al. (2009). Prevalence and geographical distribution of metachromatic leukodystrophy. **Journal of Inherited Metabolic Disease**, 32(2), 159-167.
3. Escolar, M. L., et al. (2005). Bone marrow transplantation for metachromatic leukodystrophy with mild or absent central nervous system involvement. **Neurology**, 64(2), 215-222.
4. Sessa, G., et al. (2020). A single-dose lentiviral vector gene therapy for metachromatic leukodystrophy. **Molecular Therapy**, 28(2), 432-444.
5. Food and Drug Administration (FDA) [Press Release]. (2024, March 18). FDA approves first gene therapy for children with metachromatic leukodystrophy. [https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-children-metachromatic-leukodystrophy](https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-children-metachromatic-leukodystrophy)
6. Cartier, M., et al. (2020). Gene therapy for late-onset metachromatic leukodystrophy using a lentiviral vector. **Human Gene Therapy**, 31(1-2), 39-50.

(source:internet, reference only)

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