* Fosdenopterin NULIBRY is '''cyclic pyranopterin monophosphate''' (cPMP) indicated to reduce the risk of mortality in patients with [[molybdenum cofactor deficiency]] (MoCD) Type A.
[[File:Cyclic_pyranopterin_monophosphate.svg|thumb|right|Chemical structure of cyclic pyranopterin monophosphate]]
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'''Fosdenopterin''' is a medication used in the treatment of [[molybdenum cofactor deficiency]] (MoCD) Type A, a rare genetic disorder that affects the body's ability to process certain amino acids and other substances. This condition leads to a deficiency in the molybdenum cofactor, which is essential for the activity of several important enzymes.
Fosdenopterin acts as a replacement therapy for cyclic pyranopterin monophosphate (cPMP), a precursor of the molybdenum cofactor. In patients with MoCD Type A, there is a deficiency in the enzyme molybdenum cofactor synthesis step 1 (MOCS1), which is necessary for the production of cPMP. By providing an exogenous source of cPMP, fosdenopterin helps restore the function of molybdenum-dependent enzymes.
* This medicine is indicated to reduce the risk of mortality in patients with [[molybdenum cofactor deficiency]] (MoCD) Type A'''.
=== Clinical Use ===
Fosdenopterin is specifically indicated for the treatment of patients with MoCD Type A. This condition is characterized by severe neurological damage, developmental delay, and early mortality if left untreated. The administration of fosdenopterin can help reduce the accumulation of toxic substances in the body and improve clinical outcomes.
== '''How does this medicine work?''' ==
=== Administration ===
Fosdenopterin is administered intravenously, and the dosage is typically based on the patient's weight. Treatment should be initiated as soon as possible after diagnosis to prevent irreversible neurological damage.
* Patients with MoCD Type A have mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP.
=== Side Effects ===
* '''Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin'''.
Common side effects of fosdenopterin may include fever, vomiting, and diarrhea. As with any medication, there is also a risk of allergic reactions, which can be serious.
* '''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.
== '''Who Should Not Use this medicine ?''' ==
== Related Pages ==
* [[Molybdenum cofactor deficiency]]
* [[Molybdenum cofactor]]
* [[Enzyme replacement therapy]]
* This medicine have no usage limitation
{{Medical treatment}}
== '''What drug interactions can this medicine cause?''' ==
[[Category:Drugs]]
[[Category:Genetic disorders]]
[[Category:Enzyme replacement therapy]]
== '''Is this medicine FDA approved?''' ==
* It was approved for use in the United States in 2021.
== '''How should this medicine be used?''' ==
* Start NULIBRY if known or presumed MoCD Type A. Promptly discontinue if MoCD Type A is not confirmed by genetic testing.
'''Recommended Dosage and Administration in Patients Less Than One Year of Age (by gestational age)'''
The recommended dosage regimen of NULIBRY in patients less than one year of age (by gestational age) is based on actual body weight as shown below
'''Preterm Neonates(Gestational Age Less than 37 Weeks):'''
* Initial Dosage-0.4 mg/kg once daily
* Month 1 -0.7 mg/kg once daily
* Month 3 -0.9 mg/kg once daily
'''Term Neonates (Gestational Age 37 weeks and Above)'''
* Initial Dosage-0.55 mg/kg once daily
* Month 1-0.75 mg/kg once daily
* Month 3-0.9 mg/kg once daily
'''Recommended Dosage and Administration in Patients One Year of Age or Older'''
* For patients one year of age or older, the recommended dosage of NULIBRY is 0.9 mg/kg (based on actual body weight) administered as an intravenous infusion once daily.
'''Administration'''
* NULIBRY is intended for administration by a healthcare provider. If deemed appropriate by a healthcare provider, NULIBRY may be administered at home by the patient's caregiver.
* If NULIBRY can be administered by a caregiver/patient, advise them to read the detailed instructions on the preparation, administration, storage, and disposal of NULIBRY for caregivers .
* NULIBRY is for intravenous infusion only.
* Administer with non-DEHP tubing with a 0.2 micron filter.
* Do not mix NULIBRY with other drugs (note NULIBRY is reconstituted with Sterile Water for Injection, USP).
* Do not administer as an infusion with other drugs.
* NULIBRY is given through an infusion pump at a rate of 1.5 mL per minute.
* Dose volumes below 2 mL may require syringe administration through slow intravenous push.
* Administration of NULIBRY must be completed within 4 hours of reconstitution
== '''What are the dosage forms and brand names of this medicine?''' ==
This medicine is available in fallowing doasage form:
* '''As injection: '''9.5 mg of fosdenopterin as a lyophilized powder or cake in a single-dose vial for reconstitution.
This medicine is available in fallowing brand namesː
*'''NULIBRY'''
=='''What side effects can this medication cause?'''==
The most common side effects of this medicine include:
* [[pyrexia]], viral infection
* pneumonia
* [[otitis media]]
* vomiting
* cough/sneezing
* viral upper respiratory infection
* [[gastroenteritis]]
* [[bacteremia]]
* diarrhea
==''' What special precautions should I follow?''' ==
* Limit or avoid your child's exposure or time in sunlight and artificial ultraviolet (UV) light, such as [[UVA]] or [[UVB]] [[phototherapy]].
* Your child should wear clothing, a hat, and sunglasses that protect against sun exposure.
* If your child is 6 months of age or older apply a broad spectrum sunscreen with high sun protection factor.
== '''What to do in case of emergency/overdose?''' ==
* {{overdose}}
== '''Can this medicine be used in pregnancy?''' ==
* There are no available data on NULIBRY use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.
=='''Can this medicine be used in children?'''==
* Safety and effectiveness of NULIBRY for the treatment of MoCD Type A have been established in pediatric patients starting from birth.
== '''What should I know about storage and disposal of this medication?''' ==
* Store NULIBRY frozen between -25°C and -10°C (-13°F and 14°F).
* Store the vial in its original carton to protect from light.
* For storage recommendations for the reconstituted solution.
Fosdenopterin is a medication used in the treatment of molybdenum cofactor deficiency (MoCD) Type A, a rare genetic disorder that affects the body's ability to process certain amino acids and other substances. This condition leads to a deficiency in the molybdenum cofactor, which is essential for the activity of several important enzymes.
Fosdenopterin acts as a replacement therapy for cyclic pyranopterin monophosphate (cPMP), a precursor of the molybdenum cofactor. In patients with MoCD Type A, there is a deficiency in the enzyme molybdenum cofactor synthesis step 1 (MOCS1), which is necessary for the production of cPMP. By providing an exogenous source of cPMP, fosdenopterin helps restore the function of molybdenum-dependent enzymes.
Fosdenopterin is specifically indicated for the treatment of patients with MoCD Type A. This condition is characterized by severe neurological damage, developmental delay, and early mortality if left untreated. The administration of fosdenopterin can help reduce the accumulation of toxic substances in the body and improve clinical outcomes.
Fosdenopterin is administered intravenously, and the dosage is typically based on the patient's weight. Treatment should be initiated as soon as possible after diagnosis to prevent irreversible neurological damage.
Common side effects of fosdenopterin may include fever, vomiting, and diarrhea. As with any medication, there is also a risk of allergic reactions, which can be serious.
