Nulibry NI-PASS
Research type
Research Study
Full title
A Non-interventional Post Authorisation Safety Study (PASS) of Patients with MoCD Type A Treated with NULIBRY (fosdenopterin).
IRAS ID
343090
Contact name
Hani Gabra
Contact email
Sponsor organisation
Sentynl
Clinicaltrials.gov Identifier
EMA RWD Catalogue registration, EUPAS1000000093
Duration of Study in the UK
9 years, 4 months, 29 days
Research summary
Molybdenum cofactor deficiencies (MoCD) are ultra-rare inherited metabolic disorders characterized by disruption of the metabolic pathway for production of molybdenum cofactor. Molybdenum cofactor is essential for functioning of the enzymes sulphite oxidase (SO), xanthine oxidoreductase, and aldehyde oxidase activity. There are three types of MoCD and two-thirds of MoCD patients have Type A. MoCD type A is due to a mutation in the MOCS1 gene. In MoCD Type A, the first of 4 synthetic steps in the formation of molybdenum cofactor is interrupted, and guanosine triphosphate (GTP) cannot be converted into cyclic pyranopterin monophosphate (cPMP).
NULIBRY® (fosdenopterin - a medicine developed by Sentynl Therapeutics) is the first and only treatment for children with MoCD type A. It provides an exogenous source of cPMP which is converted to molybdopterin. It is a life-long treatment administered intravenously daily. NULIBRY® has received MHRA approval in the UK. In the EU, the EMA granted market authorisation for NULIBRY® under “exceptional circumstances”. It has placed this medicine under additional monitoring and is imposing that a Non-Interventional Post-Authorisation Safety Study (NI-PASS) be conducted. The NI-PASS is to facilitate the collection of additional safety and effectiveness data on NULIBRY® use, including in patients with late-onset MoCD Type A who initiate treatment without dose titration.
The primary objective of this NI-PASS is to gather data to characterise and assess the long-term safety in patients with MoCD Type A who are prescribed and treated according to routine clinical practice.
The secondary objective of this NI-PASS is to assess the effectiveness data in all patients treated with NULIBRY.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
25/EM/0248
Date of REC Opinion
29 Oct 2025
REC opinion
Favourable Opinion