Nulibry (fosdenopterin hydrobromide)
Metabolic Disease Enzyme Replacement, MoCD Intravenous Vial (Ea)
Last reviewed · Source: First Databank (FDB) drug data
Overview
Fosdenopterin hydrobromide is a cyclic pyranopterin monophosphate (cPMP).
Fosdenopterin hydrobromide is a synthetic cyclic pyranopterin monophosphate (cPMP) that serves as a substrate replacement therapy for patients with molybdenum cofactor deficiency (MoCD) Type A.
MoCD Type A is caused by MOCS1 gene mutations that lead to deficiencies in MOCS1A/B dependent synthesis of cyclic pyranopterin monophosphate (cPMP), an intermediate substrate. Replacement of substrate with fosdenopterin provides an exogenous source of cPMP, which then undergoes conversion to molybdopterin, and subsequently, molybdenum cofactor. Molybdenum cofactor is necessary for the activation of sulfite oxidase (SOX), a molybdenum-dependent enzyme that reduces neurotoxic sulfite levels.
Following administration of single IV fosdenopterin doses of 0.075 mg/kg, 0.24 mg/kg and 0.68 mg/kg (0.08, 0.27, and 0.76 times the recommended maximum dose, respectively) in healthy adult subjects, peak plasma concentrations and AUC exhibit approximately dose-proportional increases. Plasma protein binding of fosdenopterin ranges from 6--12%. Fosdenopterin is primarily metabolized by nonenzymatic degradation processes to Compound Z, an inactive oxidative product of cPMP.
Approximately 40% of total body clearance is via the renal route. The mean elimination half-life of fosdenopterin ranges from 1.2--1.7 hours. The pharmacokinetics of fosdenopterin in pediatric patientswith MoCD Type A are simiar to that of healthy adults.
USES: This medication is used to improve survival from a certain genetic disorder (molybdenum cofactor deficiency-MoCD Type A). Fosdenopterin replaces a certain substance that is missing in people with MoCD Type A.
Generic name: fosdenopterin hydrobromide
Pronunciation: (FOS-den-OP-ter-in)
Drug class: Metabolic Disease Enzyme Replacement, MoCD
Therapeutic class: Metabolic Disease Enzyme Replacement Agents
Reference product & biosimilars
No reference, biosimilar or interchangeable associated with this drug.
Today’s intelligence on Nulibry
Indications
The following indications are listed for Nulibry:
- Molybdenum Cofactor Deficiency Type A
Also known as: Mocd Due To Mocs1 Gene Mutations, Mocod Due To Mocs1 Gene Mutations, Molybdenum Cofactor Deficiency Complementation Group A, Molybdenum Cofactor Deficiency Due To Mocs1 Gene Mutations.
Dosing
Available as fosdenopterin hydrobromide; dosage expressed in terms of fosdenopterin.
Fosdenopterin is administered by IV infusion using an infusion pump. The drug should be administeredusing non-diethylhexyl phthalate (DEHP) tubing with a 0.2 micron filter.
Fosdenopterin is intended for administration by a healthcare professional. If deemed appropriate, the drug may be administered at home by a patient orcaregiver.
If a dose of fosdenopterin is missed, administer the missed dose as soon as possible. Ensure at least 6 hours elapse between the missed dose and the next scheduled administration.
Store unopened vials between -25degreesC to -10degreesC in the original carton to protect the drug from light.
Do not mix or infuse fosdenopterin with other drugs.
Fosdenopterin is commercially available as a lyophilized powder that must be reconstituted prior to administration.
| Drug label | Dosing type | Instructions |
|---|---|---|
| Nulibry 9.5 Mg Vial | Maintenance | Infuse 0.9 mg/kg by intravenous route once daily at a rate of 1.5 mL/min. |
No dosing information is available for this tab.
Drug interactions
In vitro data indicate that fosdenopterin does not inhibit cytochrome P-450 (CYP) isoenzymes CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5, nor induces CYP1A2, CYP2B6, or CYP3A4.
Fosdenopterin is a weak substrate of, but does not inhibit, multidrug and toxin extrusion (MATE)1. Fosdenopterin is a weak inhibitor of MATE2K and organic anion transporter (OAT)1, but is not a substrate of MATE2K or OAT1. In vitro, fosdenopterin is not an inhibitor of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporter polypeptide (OATP)1B1, OATP1B3, organic cation transporter (OCT)2, or OAT3. Fosdenopterin is not a substrate of P-gp, BCRP, OATP1B1, OATP1B3, OCT2, or OAT3.
The potential for CYP or transporter-mediated drug interactions with fosdenopterin is low; no clinical drug interaction studies have been conducted with the drug.
There are no contraindication interactions for this drug.
There are no severe interactions for this drug.
There are no moderate interactions for this drug.
Contraindications
*None.
There are no contraindication contraindications for this drug.
There are no severe contraindications for this drug.
- No disease contraindications
Adverse reactions
The most common adverse effects (>25%)of fosdenopterin include catheter-related complications, pyrexia, viral infection, pneumonia, otitis media, vomiting, cough/sneezing, viral upper respiratory infection, gastroenteritis, bacteremia, and diarrhea.
More Frequent
- Bacteremia
- Pneumonia
Less Frequent
- Influenza
- Lower respiratory infection
- Seizure disorder
More Frequent
- Acute bacterial otitis media
- Cough
- Diarrhea
- Fever
- Gastroenteritis
- Sneezing
- Treatment site sequelae
- Upper respiratory infection
- Viral infection
- Vomiting
Less Frequent
- Acute abdominal pain
- Agitation
- Anemia
- Maculopapular rash
- Pain in oropharynx
- Periorbital edema
- Tonsillitis
Precautions
No enhanced messaging available.
Safety and efficacy of fosdenopterin in molybdenum cofactor deficiency (MoCD) Type A are established in pediatric patients starting at birth. Use of fosdenopterin in MoCD Type A is supported by evidence from 2 open-label and 1 observational study of 13 pediatric patientsfrom birth to 6 years of age.
No data are available to evaluate forthe risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes associated with fosdenopterin use in pregnancy. Animal reproductive studies have not been conducted with fosdenopterin.
It is not known whether fosdenopterin or its metabolites are distributed into human milk. The effects on the breast-fed infant or on the production of milk are not known.
Consider the developmental and health benefits of breast-feeding along with the mother's clinical need for the drug, and the potential for adverse effects on the breast-fed infant from the underlying maternal condition or from exposure to fosdenopterin.
Because MoCD Type A usually occurs in pediatric patients, clinical studies of fosdenopterin did not include geriatric patients.
ICD codes
| Code | Type | Description |
|---|---|---|
| Molybdenum cofactor deficiency type A | ||
| E79.8 | ICD10CM | Other disorders of purine and pyrimidine metabolism |
ICD code information from First Databank (FDB).
Formulary status
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Last reviewed · Source: First Databank (FDB) drug data
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