NULIBRY has been shown to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. It is a cyclic pyranopterin monophosphate (cPMP) which replaces a critical component the body needs to make molybdenum cofactor (MoCo).
The efficacy of NULIBRY was evaluated in clinical studies where patients received NULIBRY or an Escherichia coli-derived non-salt, anhydrous form of cPMP (recombinant cPMP or rcPMP, which has the same active moiety and the same biologic activity as NULIBRY).
In children with MoCD Type A, NULIBRY (or rcPMP) was shown to:
- Improve overall survival vs genotype-matched, untreated natural history controls
- Reduce and maintain reductions of S-sulfocysteine (SSC)
NULIBRY comes as a lyophilized powder or cake in vials that must stay frozen until ready to be used. It is delivered via cold chain by a specialty pharmacy. A medical-grade freezer is needed to help ensure NULIBRY stays within the right storage temperature range. NULIBRY must be reconstituted before it is administered as a daily intravenous (IV) infusion.
Please see Important Safety Information below.
As soon as MoCD Type A is suspected, consider NULIBRY.
NULIBRY (or rcPMP) improved survival in patients with MoCD Type A1
Efficacy assessed in a combined analysis of the 13 patients with genetically confirmed MoCD Type A (MOCS1 mutation) from Study 1 (n=8), Study 2 (n=1), and Study 3 (n=4) who received substrate replacement therapy with NULIBRY (or rcPMP).
OS in patients with MoCD Type A treated with NULIBRY (or rcPMP) vs untreated, genotype-matched natural history controls1
NULIBRY reduced the risk of death in patients with MoCD Type A1
Patients treated with NULIBRY or rcPMP had an improvement in overall survival (OS) compared to the untreated, genotype-matched, natural history controls. In these studies, NULIBRY or rcPMP reduced the risk of death by 82% compared to untreated, genotype-matched, natural history controls.
|OS in patients with MoCD Type A treated with NULIBRY (or rcPMP) versus genotype-matched untreated patients in natural history control|
|NULIBRY (or rcPMP)
|Untreated, Genotype-Matched, Natural History Controls
|Number of deaths (%)||2 (15%)||12 (67%)|
|50th Percentile (Median) Survival Time in Months (95% CI)a||NE (16, NE) months||48 (10, 99) months|
|Kaplan-Meier Survival Probablity (95% CI)
|92% (57%, 99%)
84% (49%, 96%)
|67% (40%, 83%)
55% (30%, 74%)
|Hazard Ratio for Risk of Death (95% CI)b||0.18 (0.04, 0.72)|
CI=confidence interval; NE=not estimable; rcPMP=recombinant Escherichia coli-derived cPMP.
a Quartile estimates from product-limit (Kaplan-Meier) method, with associated log-log confidence intervals.
b Based on Cox proportional hazards model regressing survival status on an indicator variable denoting treatment status. The 95% CIs are based on the modified score test statistic under the Cox model. The hazard ratio represents the risk of death in the treated patients compared to the untreated historical control patients.
NULIBRY (or rcPMP) lowered SSC levels
- Treatment with NULIBRY (or rcPMP) resulted in a reduction in the level of urinary SSC in patients with MoCD Type A, and this reduction was sustained with long-term treatment over 48 months
- Baseline level of urinary SSC was 89.8 μmol/mmol (n=1)
- Following treatment, the mean levels of urinary SSC ranged from 11.0 (±8.5) to 7.0 (±2.44) μmol/mmol from Month 3 to Month 48 (n=9)
NULIBRY demonstrated improved survival in patients with MoCD Type A.1
Common adverse reactions (>25%) reported in NULIBRY-treated patients with MoCD Type A.
NULIBRY-treated Patients (N=9)
|Catheter-related complications*||8 (89%)|
|Viral infection||5 (56%)|
|Otitis media||4 (44%)|
|Viral upper respiratory infection||3 (33%)|
*Catheter-related complications included complication associated with device, catheter site abscess, catheter site discharge, catheter site extravasation, catheter site pain, catheter site infection, catheter site inflammation, device dislocation, device leakage, device occlusion, and vascular device infection.
- Adverse reactions for the rcPMP-treated patients were similar to the NULIBRY-treated patients, except for the following additional adverse reactions that were reported in more than one patient: sepsis, oral candidiasis, varicella, fungal skin infection, and eczema
- The median exposure to NULIBRY was 4.3 years and ranged from 8 days to 5.6 years
- NULIBRY [prescribing information] https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=736aeea3-f206-454d-95d0-fd30964e8aab.