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Fosdenopterin | CAS No: 150829-29-1 | GMP-certified suppliers
A medication that reduces mortality risk in patients with molybdenum cofactor deficiency type A by restoring essential enzyme activity to prevent toxic sulfite accumulation.
Therapeutic categories
Alimentary Tract and MetabolismBiological FactorsCyclic Pyranopterin MonophosphateHeterocyclic Compounds, Fused-RingMATE 1 SubstratesMATE 2 Inhibitors
Generic name
Fosdenopterin
Molecule type
small molecule
CAS number
150829-29-1
DrugBank ID
DB16628
Approval status
Approved drug
ATC code
A16AX19
Primary indications
- Fosdenopterin is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) type A
Product Snapshot
- Fosdenopterin is an injectable peptide formulated as a lyophilized powder for intravenous solution
- It is used primarily to reduce mortality risk in patients with molybdenum cofactor deficiency type A
- Fosdenopterin holds regulatory approval in both the European Union and United States markets
Clinical Overview
Fosdenopterin is an active pharmaceutical ingredient indicated for the reduction of mortality risk in patients with molybdenum cofactor deficiency (MoCD) type A, a rare autosomal recessive metabolic disorder. MoCD type A is characterized by a deficiency in the synthesis of molybdenum cofactor, which is essential for the activity of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase, and aldehyde oxidase. The deficient activity of SOX leads to the accumulation of toxic sulfites, causing severe neurological and systemic symptoms shortly after birth.
The mechanism of action of fosdenopterin involves replacing cyclic pyranopterin monophosphate (cPMP), an intermediate in molybdenum cofactor biosynthesis. In MoCD type A, mutations in the MOCS1 gene disrupt the endogenous conversion of guanosine triphosphate to cPMP, thereby halting the production of functional molybdenum cofactor. Fosdenopterin provides the missing cPMP, enabling the synthesis of molybdenum cofactor and restoration of enzymatic activity, particularly of SOX, which detoxifies sulfites.
Pharmacodynamically, fosdenopterin therapy reduces urinary levels of S-sulfocysteine (SSC), a metabolite reflective of sulfite accumulation and a surrogate marker of treatment efficacy. Clinical evidence demonstrates that long-term administration improves survival rates, notably increasing three-year survival from approximately 55% to 84% in treated patients, a significant clinical outcome in this otherwise fatal disorder.
Safety considerations include a potential risk of phototoxicity observed in animal studies. Patients receiving fosdenopterin should minimize exposure to sunlight and artificial ultraviolet light. Protective measures such as clothing, hats, sunglasses, seeking shade, and broad-spectrum sunscreen for patients six months and older are recommended to mitigate this risk.
Fosdenopterin received marketing authorization from the U.S. FDA in February 2021 and subsequent approval by the European Medicines Agency in 2022 under exceptional circumstances for MoCD type A. It is the first and only approved therapy addressing this condition.
From a sourcing and quality perspective, fosdenopterin is a recombinant, E. coli-derived cyclic pyranopterin monophosphate. Procurement of this API requires strict adherence to regulatory standards for biologically derived materials, ensuring batch-to-batch consistency and purity to maintain clinical efficacy and safety in this rare metabolic disorder population.
The mechanism of action of fosdenopterin involves replacing cyclic pyranopterin monophosphate (cPMP), an intermediate in molybdenum cofactor biosynthesis. In MoCD type A, mutations in the MOCS1 gene disrupt the endogenous conversion of guanosine triphosphate to cPMP, thereby halting the production of functional molybdenum cofactor. Fosdenopterin provides the missing cPMP, enabling the synthesis of molybdenum cofactor and restoration of enzymatic activity, particularly of SOX, which detoxifies sulfites.
Pharmacodynamically, fosdenopterin therapy reduces urinary levels of S-sulfocysteine (SSC), a metabolite reflective of sulfite accumulation and a surrogate marker of treatment efficacy. Clinical evidence demonstrates that long-term administration improves survival rates, notably increasing three-year survival from approximately 55% to 84% in treated patients, a significant clinical outcome in this otherwise fatal disorder.
Safety considerations include a potential risk of phototoxicity observed in animal studies. Patients receiving fosdenopterin should minimize exposure to sunlight and artificial ultraviolet light. Protective measures such as clothing, hats, sunglasses, seeking shade, and broad-spectrum sunscreen for patients six months and older are recommended to mitigate this risk.
Fosdenopterin received marketing authorization from the U.S. FDA in February 2021 and subsequent approval by the European Medicines Agency in 2022 under exceptional circumstances for MoCD type A. It is the first and only approved therapy addressing this condition.
From a sourcing and quality perspective, fosdenopterin is a recombinant, E. coli-derived cyclic pyranopterin monophosphate. Procurement of this API requires strict adherence to regulatory standards for biologically derived materials, ensuring batch-to-batch consistency and purity to maintain clinical efficacy and safety in this rare metabolic disorder population.
Identification & chemistry
| Generic name | Fosdenopterin |
|---|---|
| Molecule type | Small molecule |
| CAS | 150829-29-1 |
| UNII | 4X7K2681Y7 |
| DrugBank ID | DB16628 |
Pharmacology
| Summary | Fosdenopterin is a synthetic analog of cyclic pyranopterin monophosphate (cPMP) that restores molybdenum cofactor biosynthesis in patients with molybdenum cofactor deficiency type A by bypassing the defective MOCS1 gene product. This restoration enables activation of molybdenum-dependent enzymes, notably sulfite oxidase, which detoxifies neurotoxic sulfites. The pharmacodynamic effect includes reduction of urinary sulfite metabolites, reflecting improved enzymatic function. |
|---|---|
| Mechanism of action | Molybdenum cofactor deficiency (MoCD) is a rare autosomal-recessive disorder in which patients are deficient in three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase, and aldehyde dehydrogenase. The loss of SOX activity appears to be the main driver of MoCD morbidity and mortality, as the build-up of neurotoxic sulfites typically processed by SOX results in rapid and progressive neurological damage. In MoCD type A, the disorder results from a mutation in the _MOCS1_ gene leading to deficient production of MOCS1A/B, a protein that is responsible for the first step in the synthesis of molybdenum cofactor: the conversion of guanosine triphosphate into cyclic pyranopterin monophosphate (cPMP). Fosdenopterin is an exogenous form of cPMP, replacing endogenous production and allowing for the synthesis of molybdenum cofactor to proceed. |
| Pharmacodynamics | Fosdenopterin replaces an intermediate substrate in the synthesis of molybdenum cofactor, a compound necessary for the activation of several molybdenum-dependent enzymes including sulfite oxidase (SOX). Given that SOX is responsible for detoxifying sulfur-containing acids and sulfites such as S-sulfocysteine (SSC), urinary levels of SSC can be used as a surrogate marker of efficacy for fosdenopterin. Long-term therapy with fosdenopterin has been shown to result in a sustained reduction in urinary SSC normalized to creatinine. Animal studies have identified a potential risk of phototoxicity in patients receiving fosdenopterin - these patients should avoid or minimize exposure to sunlight and/or artificial UV light. If sun exposure is necessary, use protective clothing, hats, and sunglasses, in addition to seeking shade whenever practical. Consider the use of a broad-spectrum sunscreen in patients 6 months of age or older. |
Targets
| Target | Organism | Actions |
|---|---|---|
| Molybdopterin synthase catalytic subunit | Humans | substrate |
ADME / PK
| Absorption | In healthy adult subjects, the observed C<sub>max</sub> and AUC<sub>0-inf</sub> following the intravenous administration of 0.68 mg/kg (0.76x the maximum recommended dose) were 2800 ng/mL and 5960 ng*h/mL, respectively. Both C<sub>max</sub> and AUC<sub>0-inf</sub> appear to increase proportionally with increasing doses. |
|---|---|
| Half-life | The mean half-life of fosdenopterin ranges from 1.2 to 1.7 hours. |
| Protein binding | Plasma protein binding ranges from 6 to 12%, though the specific proteins to which fosdenopterin binds have not been elucidated. |
| Metabolism | Fosdenopterin metabolism occurs mainly via non-enzymatic degradation into Compound Z, which is a pharmacologically inactive product of endogenous cyclic pyranopterin monophosphate. |
| Route of elimination | Renal clearance of fosdenopterin accounts for approximately 40% of total clearance. |
| Volume of distribution | The volume of distribution of fosdenopterin is approximately 300 mL/kg. |
| Clearance | Total body clearance of fosdenopterin ranges from 167 to 195 mL/h/kg. |
Formulation & handling
- Fosdenopterin is a small molecule API intended for intravenous injection in powder or lyophilized form, requiring reconstitution before administration.
- Due to its hydrophilic nature (LogP -2.9), formulation should ensure adequate solubility and stability in aqueous solutions.
- Handle under controlled conditions to maintain stability, avoiding exposure to moisture prior to reconstitution.
Regulatory status
| Lifecycle | The active pharmaceutical ingredient is approaching patent expiry in the United States as of January 2025, with established presence in both the US and European Union markets. Post-patent expiration, the product may experience increased generic competition influencing market dynamics. |
|---|
| Markets | EU, US |
|---|
Supply Chain
| Supply chain summary | The manufacturing and supply landscape for Fosdenopterin includes originator companies producing branded products primarily marketed under the name Nulibry in both the US and EU regions. Patent protection in the United States extends until January 31, 2025, indicating that generic competition may emerge following patent expiry. The presence of a single brand across major markets suggests a centralized originator-based supply model. |
|---|
Safety
| Toxicity | There are no data regarding overdosage of fosdenopterin. |
|---|
High Level Warnings:
- Toxicological profile of fosdenopterin is not established due to lack of overdosage data
- Handle with appropriate protective measures to prevent exposure, as safety thresholds have not been fully characterized
- Use containment and engineering controls during manufacturing to minimize occupational exposure risks
Fosdenopterin is a type of Gastrointestinal Agents
Gastrointestinal Agents belong to the pharmaceutical API category that focuses on treating disorders and ailments related to the digestive system. These agents play a crucial role in addressing various gastrointestinal conditions, such as acid reflux, ulcers, irritable bowel syndrome (IBS), and inflammatory bowel disease (IBD).
One of the key types of gastrointestinal agents is proton pump inhibitors (PPIs), which work by reducing the production of stomach acid. PPIs help in treating conditions like gastroesophageal reflux disease (GERD) and peptic ulcers. Another essential class of agents is antacids, which neutralize excessive stomach acid, providing relief from heartburn and indigestion.
Gastrointestinal agents also include antispasmodics that alleviate abdominal cramps and spasms associated with conditions like IBS. These drugs work by relaxing the smooth muscles of the digestive tract. Additionally, there are drugs categorized as laxatives that aid in relieving constipation by promoting bowel movements.
Moreover, certain gastrointestinal agents act as antiemetics, effectively reducing nausea and vomiting. These drugs are particularly useful for patients undergoing chemotherapy or experiencing motion sickness.
Pharmaceutical companies develop and manufacture a wide range of gastrointestinal agents in various forms, including tablets, capsules, suspensions, and injections. These agents are typically formulated using active pharmaceutical ingredients (APIs) and other excipients to ensure their efficacy and safety.
In conclusion, gastrointestinal agents form a vital category of pharmaceutical APIs, providing relief from digestive disorders and improving overall gastrointestinal health. The availability of diverse agents catering to different conditions ensures that patients can receive targeted treatment for their specific gastrointestinal needs.
