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Nulibry (FOSDENOPTERIN)
Nulibry works by providing a functional molybdenum cofactor to cells, bypassing the genetic defect that causes molybdenum cofactor deficiency type A.
At a glance
| Generic name | FOSDENOPTERIN |
|---|---|
| Sponsor | Sentynl Theraps Inc |
| Drug class | Cyclic Pyranopterin Monophosphate |
| Target | Molybdenum cofactor biosynthesis protein 1 |
| Modality | Small molecule |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2021 |
Mechanism of action
Patients with MoCD Type have mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites.
Approved indications
- Molybdenum cofactor deficiency type A
Common side effects
- Catheter-related complications
- Pyrexia
- Viral infection
- Pneumonia
- Otitis Media
- Vomiting
- Cough/Sneezing
- Upper viral respiratory infection
- Gastroenteritis
- Diarrhea
- Bacteremia
- Abdominal pain
Key clinical trials
- Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A (PHASE2,PHASE3)
- Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP (PHASE2)
- A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
- Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A (PHASE1,PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Nulibry CI brief — competitive landscape report
- Nulibry updates RSS · CI watch RSS
- Sentynl Theraps Inc portfolio CI