fosdenopterin
Orphan Drug Cold Chain RequiredFDA Approved (2021), Unregistered in India
Description
Fosdenopterin is the first and only approved treatment for molybdenum cofactor deficiency type A (MoCD Type A), an extremely rare and often fatal genetic disorder. It works by providing an exogenous source of cyclic pyranopterin monophosphate (cPMP), which is deficient in patients. Early treatment is critical for survival and neurological outcomes.
Indications & Therapeutic Use
Molybdenum cofactor deficiency type A (MoCD Type A)
Linked Diseases:
Global Availability (3 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — | |
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
fosdenopterin
| Generic Name | fosdenopterin |
| Brands | 1 brand available |
| Active Ingredient | fosdenopterin hydrobromide |
| Drug Class | Molybdenum cofactor deficiency type A (MoCD Type A) |
| Manufacturer | Origin Biosciences |
| Dosage Forms | Intravenous infusion, 0.44mg/mL concentrate for solution |
| Medical Code | A16AX14 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 30 days |
| Reg. Status | FDA Approved (2021), Unregistered in India |
| Clinical Trial | NCT02047461 |
| Countries | 3 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations4 Validated Nodes