Nulibry
fosdenopterin
Table of contents
Overview
Nulibry is a medicine used to treat patients with molybdenum cofactor deficiency (MoCD) type A.
MoCD type A is an inherited disease where patients do not have enough of a substance called 'molybdenum cofactor', a molecule needed for the production of certain enzymes. Without these enzymes, the toxic chemical sulfite builds up in the brain causing brain damage.
In the 'type A' form of the disease, the absence of molybdenum cofactor is due to patients lacking a substance called 'cyclic pyranopterin monophosphate (cPMP)’, which the body needs to make molybdenum cofactor.
MoCD type A is rare, and Nulibry was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 September 2010. Further information on the orphan designation can be found here.
Nulibry contains the active substance fosdenopterin.
-
List item
Nulibry : EPAR - Medicine Overview (PDF/125.83 KB)
First published: 29/09/2022
EMEA/H/C/005378 -
-
List item
Nulibry : EPAR - Risk management plan (PDF/921.04 KB)
First published: 29/09/2022
Authorisation details
Product details | |
---|---|
Name |
Nulibry
|
Agency product number |
EMEA/H/C/005378
|
Active substance |
fosdenopterin hydrobromide dihydrate
|
International non-proprietary name (INN) or common name |
fosdenopterin
|
Therapeutic area (MeSH) |
Metal Metabolism, Inborn Errors
|
Anatomical therapeutic chemical (ATC) code |
A16AX19
|
Additional monitoring |
This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring. |
Orphan |
This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation. |
Publication details | |
---|---|
Marketing-authorisation holder |
Zydus France S.A.S.
|
Revision |
1
|
Date of issue of marketing authorisation valid throughout the European Union |
15/09/2022
|
Contact address |
Zydus France S.A.S. |
Product information
03/02/2023 Nulibry - EMEA/H/C/005378 - T/0002
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
Pharmacotherapeutic group
Other alimentary tract and metabolism products
Therapeutic indication
NULIBRY is indicated for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.