Nirogacestat hydrobromide - Generic Drug Details

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

Nirogacestat hydrobromide is the generic ingredient in one branded drug marketed by Springworks and is included in one NDA. There are thirty patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Nirogacestat hydrobromide has one hundred and twenty-four patent family members in forty-five countries.

One supplier is listed for this compound.

Summary for nirogacestat hydrobromide

International Patents:	124
US Patents:	30
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Applications:	122
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for nirogacestat hydrobromide
DailyMed Link:	nirogacestat hydrobromide at DailyMed

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

Constraining patent/regulatory exclusivity:

FOR ADULT PATIENTS WITH PROGRESSING DESMOID TUMORS WHO REQUIRE SYSTEMIC TREATMENT

Dosage:

TABLET;ORAL

^*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 nirogacestat hydrobromide

Drug Class	Gamma Secretase Inhibitor
Mechanism of Action	Cytochrome P450 2C19 Inducers Cytochrome P450 3A Inhibitors Gamma Secretase Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for nirogacestat hydrobromide

L01XX	Other antineoplastic agents
L01X	OTHER ANTINEOPLASTIC AGENTS
L01	ANTINEOPLASTIC AGENTS
L	Antineoplastic and immunomodulating agents

US Patents and Regulatory Information for nirogacestat hydrobromide

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-002	Apr 4, 2024		RX	Yes	No			Y
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-002	Apr 4, 2024		RX	Yes	No			Y
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-001	Nov 27, 2023		RX	Yes	No
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-002	Apr 4, 2024		RX	Yes	No				Y
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-001	Nov 27, 2023		RX	Yes	No
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-003	Apr 4, 2024		RX	Yes	Yes
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-001	Nov 27, 2023		RX	Yes	No				Y

Expired US Patents for nirogacestat hydrobromide

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-001	Nov 27, 2023
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-002	Apr 4, 2024
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-002	Apr 4, 2024
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-003	Apr 4, 2024
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-003	Apr 4, 2024
Springworks	OGSIVEO	nirogacestat hydrobromide	TABLET;ORAL	217677-001	Nov 27, 2023

International Patents for nirogacestat hydrobromide

Country	Patent Number	Title	Estimated Expiration
Croatia	P20080375
Argentina	049875	COMPUESTOS DE IMIDAZOL PARA EL TRATAMIENTO DE TRASTORNOS NEURODEGENERATIVOS
Peru	20051155	COMPUESTOS DE IMIDAZOL PARA EL TRATAMIENTO DE TRASTORNOS NEURODEGENERATIVOS
World Intellectual Property Organization (WIPO)	2023225283
World Intellectual Property Organization (WIPO)	2023039475
European Patent Office	4426294	COMPOSITIONS ET TRAITEMENTS À BASE DE NIROGACESTAT (COMPOSITIONS AND TREATMENTS WITH NIROGACESTAT)
China	114555562

Market Dynamics and Financial Trajectory for Nirogacestat Hydrobromide

Last updated: July 29, 2025

Introduction

Nirogacestat hydrobromide, a selective gamma-secretase inhibitor, has garnered significant attention within oncology therapeutics due to its potential in treating desmoid tumors and other tumor types. Developed by PharmaCyte, this drug faces evolving market dynamics driven by clinical progress, regulatory pathways, competitive landscape, and commercial considerations. Analyzing its market trajectory necessitates understanding current therapeutic needs, pipeline positioning, and projected financial outcomes.

Therapeutic Indication and Clinical Development Status

Nirogacestat operates primarily within the niche of desmoid tumor treatment, an ultra-rare, non-metastasizing fibrous neoplasm characterized by aggressive local invasion and high recurrence rates. The drug's mechanism of inhibiting gamma-secretase affects the Notch signaling pathway—a critical driver in tumorigenesis.

Recent clinical trials, notably the Phase II (DeFi) trial, showed promising efficacy signals. Results demonstrated significant tumor responses and manageable safety profiles, propelling the drug into advanced development stages. Regulatory agencies like the FDA have granted Orphan Drug Designation, highlighting its potential to address unmet medical needs.

Market Landscape and Competitive Dynamics

1. Unmet Medical Needs in Desmoid Tumors

Desmoid tumors pose treatment challenges; standard modalities include surgery, radiotherapy, and systemic agents such as NSAIDs, hormonal therapy, and chemotherapy, with limited efficacy and high recurrence. The orphan designation and positive trial outcomes position nirogacestat as a pioneering targeted therapy with a compelling market entry.

2. Competition and Alternatives

Currently, few targeted drugs exist for desmoid tumors. Sorafenib and pazopanib, anti-angiogenic agents, have shown some activity but lack formal approval specifically for this indication. The limited competition enhances nirogacestat’s market potential but also necessitates rigorous clinical validation.

3. Broader Oncology Applications

Beyond desmoid tumors, gamma-secretase inhibitors have potential utility across other solid tumors and neuroendocrine cancers; however, this exploration remains at preclinical or early clinical stages. Diversification can de-risk revenue streams but introduces competitive and regulatory challenges.

Regulatory and Market Access Considerations

1. Accelerated Approval Pathways

Given the life-altering nature of desmoid tumors and regulatory incentives like Orphan Drug and Fast Track designations, PharmaCyte might pursue accelerated approval, expediting market entry and initial revenue generation.

2. Pricing and Reimbursement Landscape

Orphan drugs typically command high-price premiums reflective of clinical benefit and rarity. Payer reimbursement strategies will largely depend on demonstrated efficacy, safety, and quality-of-life improvements.

3. Manufacturing and Supply Chain

Complex manufacturing processes for biologically derived agents can influence margins. Ensuring robust supply chains and scalable production remains vital for market success.

Financial Trajectory and Revenue Projections

1. Revenue Potential

Based on clinical data and market size estimates, the global desmoid tumor incidence is approximately 2-4 cases per million annually, translating into a niche market. Assuming successful commercialization and premium pricing (e.g., $100,000 – $150,000 per treatment course), potential peak sales could range from $200 million to $500 million globally.

2. Development Costs and Investment

Investments encompass clinical trial phases, regulatory filings, manufacturing setup, and commercialization activities. Early-phase development costs could range from $50 million to $100 million, with additional capital for late-phase trials and market access.

3. Return on Investment (ROI)

Timely regulatory approval and successful commercialization could yield substantial ROI within five years, contingent on approvals, pricing strategies, and market uptake. The drug’s orphan designation offers exclusivity for seven years post-approval, providing a competitive moat to recoup investments.

4. Risk Factors Impacting Financial Trajectory

Key risks include clinical trial failure, regulatory delays, pricing pressures, potential competition, and market acceptance. External factors like reimbursement policy shifts and manufacturing disruptions further influence financial outcomes.

Market Expansion and Future Opportunities

While initial focus centers on desmoid tumors, the therapeutic platform’s versatility could extend to other gamma-secretase–associated indications, such as multiple myeloma or Alzheimer's disease, pending successful trials. Strategic collaborations and licensing agreements could facilitate diversification.

Conclusion

Nirogacestat hydrobromide’s market dynamics hinge on clinical validation, regulatory pathways, and niche market conditions. Its potential to transform desmoid tumor management, combined with high unmet needs and favorable regulatory designations, underpin a promising financial trajectory. However, success ultimately depends on clinical efficacy, regulatory approval, market access, and the competitive landscape.

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Key Takeaways

• Market Opportunity: Nirogacestat targets a rare but high-need indication, offering a unique market position with minimal competition.
• Clinical Validation: Robust Phase II results support further development; positive trial outcomes are crucial for regulatory approval.
• Revenue Potential: Peak sales could reach hundreds of millions annually, driven by premium pricing in the orphan drug space.
• Risks and Challenges: Clinical, regulatory, and market risks necessitate vigilant risk mitigation strategies.
• Expansion Prospects: Future indications could broaden its market and enhance long-term financial sustainability.

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FAQs

1. What is the primary therapeutic target of nirogacestat hydrobromide?
It selectively inhibits gamma-secretase, impacting the Notch signaling pathway implicated in tumor growth, especially in desmoid tumors.

2. What regulatory designations support its development?
The drug has received Orphan Drug and Fast Track designations from the FDA, facilitating expedited review and market exclusivity.

3. How does nirogacestat compare to existing treatments for desmoid tumors?
Currently, no targeted approval exists specifically for desmoid tumors; treatments like surgery and radiotherapy often have high recurrence. Nirogacestat shows promise as a systemic, targeted therapy with potentially superior efficacy and safety.

4. What are the main revenue drivers for nirogacestat?
High unmet medical need, orphan drug status, premium pricing, and limited competition serve as primary revenue drivers.

5. What are the critical factors influencing the drug's market success?
Successful clinical trial outcomes, timely regulatory approval, market acceptance, reimbursement strategies, and manufacturing scalability are pivotal.

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Sources
[1] Clinical trial results and regulatory filings.
[2] Epidemiological data for desmoid tumors.
[3] Industry reports on orphan drug pricing and market size estimates.
[4] Strategic analysis on gamma-secretase inhibitors.