Friday, July 10, 2026

Drug profile · INN

autologous cd34+ cells encoding arsa gene

autologous cd34+ cells encoding arsa gene (LIBMELDY) is a arsa exogenous gene.

EMA: approved Nervous system (N07) N07
Patents linked 0
Pipeline programs 3
News articles 0
Drug details — autologous cd34+ cells encoding arsa gene
Class
Nervous system (N07)
Route
Patents
0
Programs
3
Data quality
0.83

Quick answer

autologous cd34+ cells encoding arsa gene (LIBMELDY) is a arsa exogenous gene.

Key facts

INN autologous cd34+ cells encoding arsa gene
Brand LIBMELDY
Mechanism ARSA exogenous gene
Target ARSA
Therapeutic class Nervous system (N07)
ATC code N07

Mechanism of action

ARSA exogenous gene

Primary target: ARSA

Approval history

Authority Status Date
EMA approved

Pipeline programs

  1. Libmeldy 2-10 x 10^6 cells/mL dispersion for infusion Phase 3 · ORCHARD THERAPEUTICS · Metachromatic Leukodystrophy (MLD) is an autosomal recessive
  2. Libmeldy 2-10 x 10^6 cells/mL dispersion for infusion Phase 3 · ORCHARD THERAPEUTICS · Metachromatic Leukodystrophy (MLD)
  3. Libmeldy 2-10 x 10^6 cells/mL dispersion for infusion Phase 2 · ORCHARD THERAPEUTICS · Metachromatic Leukodystrophy (MLD)

Related & competitor drugs

  1. aripiprazole Nervous system (N05)
  2. brexpiprazole Nervous system (N05)
  3. brivaracetam Nervous system (N03)
  4. bupivacaine Nervous system (N01)
  5. buprenorphine Nervous system (N07)
  6. caffeine citrate Nervous system (N06)

Frequently asked questions

What is autologous cd34+ cells encoding arsa gene?

autologous cd34+ cells encoding arsa gene (LIBMELDY) is a arsa exogenous gene.

What is the brand name for autologous cd34+ cells encoding arsa gene?

LIBMELDY is a marketed brand name for autologous cd34+ cells encoding arsa gene. Check FDA Drugs@FDA for the current US label holder and prescribing information.