Fosdenopterin
Fosdenopterin (Nulibry) is a substrate replacement therapy used to treat Molybdenum Cofactor Deficiency Type A (MoCD Type A), a rare genetic metabolic disorder. It works by providing an exogenous source of cPMP, which the body converts to molybdopterin, a critical cofactor for enzymes involved in sulfur amino acid metabolism. This medication is specifically indicated for patients with MoCD Type A caused by MOCS1 gene mutations.
Fosdenopterin is a substrate replacement therapy for rare Molybdenum Cofactor Deficiency Type A that provides the missing cPMP needed for molybdopterin synthesis.
Key facts at a glance
- ATC
- A16AX19
- RxNorm
- 2531288
- Generic
- Fosdenopterin
- Class
- atc-a16
- Brands
- Nulibry
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treatment of Molybdenum Cofactor Deficiency Type A (MoCD Type A), a rare autosomal recessive genetic metabolic disorder caused by mutations in the MOCS1 gene that affects sulfur amino acid metabolism
Acts as substrate replacement therapy by providing an exogenous source of cyclic pyranopterin monophosphate (cPMP), which is converted to molybdopterin, restoring function of molybdopterin-dependent enzymes
Addresses the underlying metabolic deficiency rather than just treating symptoms; specific to MoCD Type A and not indicated for other forms of MoCD or other metabolic conditions
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy