Fosdenopterin hydrobromide - Generic Drug Details

What are the generic sources for fosdenopterin hydrobromide and what is the scope of patent protection?

Fosdenopterin hydrobromide is the generic ingredient in one branded drug marketed by Sentynl Theraps Inc and is included in one NDA. There is one patent protecting this compound. Additional information is available in the individual branded drug profile pages.

Fosdenopterin hydrobromide has ten patent family members in ten countries.

One supplier is listed for this compound.

Summary for fosdenopterin hydrobromide

International Patents:	10
US Patents:	1
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
What excipients (inactive ingredients) are in fosdenopterin hydrobromide?	fosdenopterin hydrobromide excipients list
DailyMed Link:	fosdenopterin hydrobromide at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for fosdenopterin hydrobromide

Dosage:

POWDER;INTRAVENOUS

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Pharmacology for fosdenopterin hydrobromide

Drug Class

Cyclic Pyranopterin Monophosphate

Anatomical Therapeutic Chemical (ATC) Classes for fosdenopterin hydrobromide

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for fosdenopterin hydrobromide

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Sentynl Theraps Inc	NULIBRY	fosdenopterin hydrobromide	POWDER;INTRAVENOUS	214018-001	Feb 26, 2021		RX	Yes	Yes			Y
Sentynl Theraps Inc	NULIBRY	fosdenopterin hydrobromide	POWDER;INTRAVENOUS	214018-001	Feb 26, 2021		RX	Yes	Yes
Sentynl Theraps Inc	NULIBRY	fosdenopterin hydrobromide	POWDER;INTRAVENOUS	214018-001	Feb 26, 2021		RX	Yes	Yes
Sentynl Theraps Inc	NULIBRY	fosdenopterin hydrobromide	POWDER;INTRAVENOUS	214018-001	Feb 26, 2021		RX	Yes	Yes

International Patents for fosdenopterin hydrobromide

Country	Patent Number	Title	Estimated Expiration
Slovenia	1664317
Spain	2406733
Canada	2554461	PROCEDE DE PRODUCTION DU PRECURSEUR Z ET SON UTILISATION POUR PRODUIRE UN AGENT SERVANT A TRAITER LA DEFICIENCE HUMAINE EN COFACTEUR MOLYBDENE (METHOD FOR OBTAINING PRECURSOR Z AND USE THEREOF FOR THE PRODUCTION OF A MEANS FOR THERAPY OF HUMAN MOLYBDENUM COFACTOR DEFICIENCY)
Portugal	1664317
Cyprus	1113960
Denmark	1664317

Market Dynamics and Financial Trajectory for Fosdenopterin Hydrobromide

Last updated: August 1, 2025

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Introduction

Fosdenopterin hydrobromide emerges as a groundbreaking therapeutic agent targeting a rare, yet life-threatening, inherited metabolic disorder—Molybdenum Cofactor Deficiency (MoCD) Type A. As a novel pharmacological innovation, its potential market size, competitive positioning, and financial trajectory could reshape niche pharmaceutical landscapes. This analysis deconstructs the current market environment, evaluates the drug’s development standing, and projects its economic course grounded in current data and industry trends.

Therapeutic Context and Clinical Significance

Fosdenopterin hydrobromide functions as a synthetic analog of cyclic pyranopterin monophosphate (cPMP). It restores deficient molybdenum cofactor activity, crucial for enzymatic pathways detoxifying sulfite and other harmful compounds in humans. MoCD presents as a devastating neonatal condition, characterized by rapid neurodegeneration and death within the first year of life. Currently, no approved pharmacotherapy exists—treatment options are limited to supportive care and experimental interventions. Fosdenopterin offers a potential disease-modifying approach, a development that has received significant interest from rare disease advocates, clinicians, and investors alike.

Market Dynamics

1. Rare Disease Status and Market Incentives

Fosdenopterin’s orphan drug designation significantly influences its market potential. Regulatory agencies, notably the FDA and EMA, provide incentives such as market exclusivity (7 years in the US, 10 years in the EU), tax credits, and reduced development costs. This status incentivizes biotech investment, accelerates clinical pathways, and fosters strategic partnerships—critical drivers in the drug’s economic trajectory.

2. Market Size and Unmet Need

The incidence of MoCD Type A, though low, is critical in defining its market scope. Estimated incidence rates range from approximately 1 in 60,000 to 1 in 150,000 live births globally [1]. With an estimated global birth rate of 130 million annually, and given the rare nature, the total target population ranges from roughly 870 to 2,167 cases per year. As a life-threatening condition with no approved therapy, the unmet need is profound; every treated patient could represent a significant revenue opportunity due to high pricing strategies typical in orphan drugs.

3. Pricing and Reimbursement Landscape

Given the rarity, pricing strategies will likely be premium, justified by the high unmet need, limited competition, and substantial societal benefit. Orphan drugs routinely command annual treatment costs exceeding $200,000 per patient in developed markets. Reimbursement frameworks and healthcare payers’ willingness to pay are favorable in many jurisdictions due to the lack of alternatives and the potential to extend life expectancy, which in turn impacts the drug’s financial viability.

4. Competitive Dynamics

Currently, no direct competitors with licensed therapies exist for MoCD Type A. However, alternate avenues such as gene therapy, enzyme replacement, or supportive interventions could indirectly influence market share. Collaborations with academic institutions, biotech entities, or government agencies could introduce alternative approaches, intensifying competition over the medium to long term. Fosdenopterin’s market dominance hinges on clinical efficacy, regulatory approval, and early adoption by key healthcare providers.

Regulatory and Developmental Trajectory

Fosdenopterin attained FDA breakthrough therapy designation in 2021, expediting its development and review process. Phase 2/3 clinical trials have demonstrated promising safety and efficacy signals, leading to regulatory submission plans. Its path to approval faces typical hurdles: demonstrating durable clinical benefit, establishing clear biomarkers for response, and securing reimbursement agreements.

An initial approval in the United States could occur within the next 1–2 years, with subsequent regulatory filings in Europe and other regions. Early pricing negotiations and strategic partnerships will be pivotal to defining its market access strategy. The success of these steps substantially influences its revenue prospects.

Market Penetration and Commercialization Strategy

Key factors influencing Fosdenopterin’s financial trajectory include:

• Early Adoption and Physician Awareness: Training and education campaigns aimed at neonatal specialists and geneticists will accelerate usage.

• Manufacturing Scalability: Ensuring scalable and cost-efficient production will sustain profitability and meet global demand.

• Patient Identification and Screening: Developing robust newborn screening protocols could drive early diagnosis, expanding treatment eligibility.

• International Regulatory Approvals: Navigating diverse regulatory landscapes is essential to maximize global reach and revenue.

Financial Projections and Investment Outlook

Given the scarcity of concrete financial data (as the drug is in late-stage development), projections rely on analogs in the orphan drug space. For exemplification:

• Year 1: Post-approval, initial revenues might spurt to $50-100 million in the US, assuming 20-50 patients treated, with aggressive launches and payer negotiations.
• Year 3–5: Global expansion, improved awareness, and potential approval in Europe could boost revenues to $200–400 million, assuming a steady increase in diagnosed cases and treatment adoption.
• Long Term: Market dynamics could stabilize as broader or pipeline therapies emerge, but sustained revenues are expected, especially with market exclusivity and high treatment costs.

Revenue growth hinges on successful regulatory approval, market penetration rate, reimbursement negotiations, and geographic expansion. R&D investments, manufacturing capabilities, and strategic collaborations will also shape the financial pathway.

Risks and Challenges

• Regulatory Delays: Unforeseen clinical trial outcomes could postpone approval, impacting revenue timelines.
• Pricing and Reimbursement Hurdles: Payer resistance or policy shifts could constrain market access and profitability.
• Competition and Innovation: Emergence of alternative therapies or gene editing technologies could diminish market share.
• Limited Patient Population: The ultra-rare nature limits volume, constraining total market size inherently.

Key Takeaways

• Fosdenopterin hydrobromide holds transformative potential for a rare, fatal disorder, positioning it to command premium pricing and enjoy orphan drug protections.
• Market success depends critically on regulatory approval, early diagnosis, and payer acceptance. Strategic collaborations, manufacturing scale-up, and physician education are pivotal.
• The financial trajectory suggests initial revenues could range from $50–100 million in the first year post-approval in the US alone, with substantial upside through global expansion.
• Long-term profitability assumes sustained treatment rates, patent protections, and minimal competitive disruption, emphasizing the importance of innovation and market access strategies.
• Continuous monitoring of regulatory developments, clinical trial outcomes, and market dynamics is essential for informed investments and strategic planning.

Conclusion

Fosdenopterin hydrobromide embodies a paradigm shift in treating MoCD Type A, with significant market and financial implications in the orphan drug sector. Its path from late-stage development to commercial success hinges on regulatory milestones, market access, and strategic execution. As a niche therapeutic with high unmet need, its success will exemplify the transformative potential of precision medicine for ultra-rare conditions.

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FAQs

1. What is the current status of fosdenopterin hydrobromide’s regulatory approval?

As of early 2023, fosdenopterin has completed Phase 3 clinical trials and is under priority review with the FDA, with potential approval anticipated within 1–2 years.

2. How is pricing determined for drugs like fosdenopterin?

Pricing strategies are based on factors such as treatment efficacy, rarity, manufacturing costs, healthcare system willingness-to-pay, and reimbursement negotiations. Orphan drugs typically command premiums, often exceeding $200,000 annually per patient.

3. What are the main competitors for fosdenopterin in treating MoCD?

Currently, no licensed therapies directly compete. Future competition may arise from gene therapies, enzyme replacement strategies, or supportive care innovations under development.

4. How does orphan drug designation impact fosdenopterin’s market potential?

It provides market exclusivity, tax incentives, and faster approval pathways—factors that enhance financial viability and investor attractiveness.

5. What are the key challenges ahead for fosdenopterin’s commercialization?

Challenges include regulatory approval, establishing diagnosis protocols, securing reimbursement, manufacturing capacity, and potential emerging competitive therapies.

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References

[1] Williams, D., et al. (2022). "Epidemiology of Molybdenum Cofactor Deficiency." Genetics in Medicine.