Details Libmeldy

Data as extracted from Orphan Designation Register

Product (OD)

Libmeldy

PL number (OD)

PLGB 49055/0002

OD Designation ID (OD)

OD1

Active substance (OD)

Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene

Data as extracted from SMPC. (PS: More information at bottom of the page)

Product (SMPC)

PL Number (SMPC)

Composition/ Active Substance (SMPC)

Dose form (SMPC)

Marketing Authorisation holder details (SMPC)

IDMP View

Structured regulatory data for this product mapped to the IDMP entity hierarchy (ISO 11615) — Marketing Authorisation, Medicinal Product, and Administrable Product.
Note: Wherever a type is listed as "Equivalent Term not known", we dont have the information available in public domain. However, this data is usually available in eAF (or equivalent) form and is stored by most regulators as strucutured data - albeit not always in same hierarchy as IDMP.

No IDMP data is linked to this authorisation number.

AI match to EMA Substance Dictionary

We analysed the SMPC composition text and searched for the closest equivalent in EMA's published substance dictionary. The suggested match and supporting details are shown below.

SMPC composition / active substance

Matched EMA substance

EMA SMS ID

Confidence

Higher confidence means the match is more likely to be correct.

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FDA Approved Products —

Products approved by the FDA containing the matched EMA substance, sourced from OpenFDA Drugs@FDA.

No EMA substance match available — FDA lookup skipped.

Further information for:

Select a section below to read the detailed content extracted from the SMPC / Orphan Designation register.

OD Indication (OD)

ibmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity: in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline

Full Designation Number(OD)

PLGB 49055/0002/OD1

Orphan Condition(OD)

Metachromatic leukodystrophy

All Indications (SMPC)

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Pregnancy/Lactation (SMPC)

Driving/Machines (SMPC)

Undesirable effects (SMPC)

Overdose (SMPC)

Shelf life (SMPC)

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Container description (SMPC)

Handling/Disposal (SMPC)

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