Atidarsagene autotemcel

Overview

Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy comprising genetically modified autologous CD34+ enriched hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic leukodystrophy (MLD).

Indication

Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity in children with:

Associated Conditions

Metachromatic Leukodystrophy (MLD)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor

No clinical trials found

No clinical trials found for this drug

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
LENMELDY	Orchard Therapeutics (Europe) Ltd	83222-0200	INTRAVENOUS	11800000 1 in 1 1	8/12/2025

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Libmeldy	Orchard Therapeutics (Netherlands) BV,Prins Bernhardplein 200,1097 JB Amsterdam,The Netherlands	Authorised	12/17/2020
Libmeldy	Orchard Therapeutics (Netherlands) BV,Prins Bernhardplein 200,1097 JB Amsterdam,The Netherlands	Authorised	12/17/2020
Libmeldy	Orchard Therapeutics (Netherlands) B.V.,Bargelaan 200,2333 CW Leiden,Netherlands	Authorised	12/17/2020

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
No Health Canada approvals found for this drug.

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

U.S. Drug Prices Double as Pharmaceutical Industry Shifts Focus to Rare Diseases

3 months ago

The median annual list price for newly launched pharmaceuticals in the U.S. has more than doubled to $370,000 in 2024 compared to $180,000 in 2021, according to a Reuters analysis.

Orchard Therapeutics' Lenmeldy Shows Long-Term Efficacy in Treating Metachromatic Leukodystrophy, NEJM Study Reveals

3 months ago

• Lenmeldy (atidarsagene autotemcel), Orchard Therapeutics' hematopoietic stem cell gene therapy, demonstrated significant extension of life expectancy and preservation of motor and cognitive function in children with early-onset metachromatic leukodystrophy. • The New England Journal of Medicine publication details over 12 years of follow-up data with more than 250 patient-years of experience, showing durable efficacy in pre-symptomatic and early-symptomatic patients. • Orchard Therapeutics will present six studies at the upcoming American Society of Gene and Cell Therapy meeting, highlighting their expanding portfolio of hematopoietic stem cell gene therapies for rare neurometabolic diseases.

FDA Approves Lenmeldy: $4.25 Million Gene Therapy for Rare Childhood Disease MLD

4 months ago

• Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) has become the first FDA-approved treatment for metachromatic leukodystrophy (MLD), a rare genetic disorder that causes rapid neurological deterioration in children. • At $4.25 million per one-time treatment, Lenmeldy now holds the title of the most expensive medicine in the United States, slightly higher than its European price of $3.7 million where it's marketed as Libmeldy. • Clinical trials showed remarkable results with all 37 treated children surviving to age six (compared to 58% in control groups), with 71% able to walk independently and 85% maintaining normal language and cognitive function.

FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture

5 months ago

Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.

FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months

6 months ago

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

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