ELZONRIS Drug Profile

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Summary for Tradename: ELZONRIS

High Confidence Patents:	0
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for ELZONRIS

Recent Clinical Trials for ELZONRIS

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
Jonsson Comprehensive Cancer Center	Phase 1
Stemline Therapeutics, Inc.	Phase 1
Therapeutic Advances in Childhood Leukemia Consortium	Phase 1

See all ELZONRIS clinical trials

Pharmacology for ELZONRIS

Mechanism of Action	CD123 Interactions
Established Pharmacologic Class	CD123 Interaction Cytotoxin
Chemical Structure	Cytotoxins

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

DrugPatentWatch analysis and company disclosures

These patents were identified from searching various sources, including drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for ELZONRIS Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for ELZONRIS Derived from DrugPatentWatch Analysis and Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for ELZONRIS Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for the Biologic Drug: ELZONRIS

Introduction
ELZONRIS (tagraxofusp-erzs), developed by Stemline Therapeutics (acquired by Moderna), is a biologic therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive hematologic malignancy. The drug’s unique mechanism as a CD123-targeted immunotherapy has positioned it within a niche but critical segment of oncology therapeutics. Analyzing ELZONRIS’s market dynamics and financial trajectory offers insights into its growth potential, competitive positioning, and impact on the broader biologic and hematology landscapes.

Market Overview and Therapeutic Landscape
BLDCN represents a formidable challenge with limited treatment options, underscoring ELZONRIS’s clinical significance. Prior to its approval in 2018, BPDCN management was predominantly palliative, relying on chemotherapy with modest success. ELZONRIS introduced a paradigm shift, providing a targeted immunotherapeutic approach tailored for this indication. The drug’s mechanism involves a fusion protein combining interleukin-3 with a truncated diphtheria toxin, directing cytotoxic activity specifically to cells overexpressing CD123.

The orphan status and accelerated approval pathway initially facilitated ELZONRIS’s market entry, but commercial adoption remains influenced by disease rarity, limited patient population, and competition from emerging therapies. The landscape comprises traditional chemotherapies, hematopoietic stem cell transplantation, and emerging immunotherapeutics, such as bispecific antibodies and CAR-T cells targeting similar pathways.

Market Dynamics Influencers

Orphan Drug Designation and Market Incentives
ELZONRIS benefits from orphan drug designation, providing market exclusivity through 2028 in the U.S., along with tax credits, fee waivers, and potential faster regulatory review processes. This exclusivity is crucial in safeguarding revenue streams against generic or biosimilar entries for related biologics, though not for ELZONRIS specifically, given its tailored nature.

Clinical and Regulatory Developments
Recent clinical trials have explored ELZONRIS’s utility beyond BPDCN, including preliminary investigations into other CD123-expressing hematologic cancers. Demonstrating efficacy enrolls the drug in broader therapeutic dialogues, potentially expanding its market scope. Regulatory programs like FDA Breakthrough Therapy designation for some indications expedite development and approval, further influencing market trajectory.

Market Penetration and Geographic Expansion
Despite orphan drug status, access to ELZONRIS outside the U.S. remains limited, hampered by pricing, reimbursement challenges, and manufacturing capacities. Moderna's acquisition of Stemline and the subsequent integration of ELZONRIS into its portfolio set the stage for international distribution, subject to regional regulatory landscapes.

Pricing and Reimbursement Strategies
ELZONRIS’s high manufacturing complexity, coupled with the dire prognosis of BPDCN, justifies premium pricing. In the U.S., the drug’s cost has ranged around $244,000 per treatment course, positioning it in a high-value segment. Reimbursement negotiations with payers shape real-world utilization; coverage varies based on regional healthcare policies.

Competitive and Emerging Therapies
Ongoing research into CD123-targeted bispecific antibodies, immunotoxins, and CAR-T cells challenges ELZONRIS’s market share. While these agents may offer advantages such as reduced toxicity or enhanced efficacy, their clinical validation remains in early phases, providing a window for ELZONRIS’s established presence.

Financial Trajectory and Revenue Outlook

Last updated: September 29, 2025

Historical Revenue Performance
Since FDA approval in 2018, sales of ELZONRIS have shown modest initial uptake, influenced by the rarity of BPDCN and limited awareness among clinicians. According to publicly available data, cumulative U.S. sales amounted to approximately $90-100 million within the first few years, with incremental growth driven by increased recognition and expanded clinical use.

Growth Drivers

Market Penetration in Core Indication: As prescribing physicians become more familiar with ELZONRIS, treatment rates are expected to increase modestly. The rarity of BPDCN constrains absolute sales volume but sustains high per-patient revenue.
International Expansion: Entry into European and Asian markets hinges on regulatory approvals, which are currently in progress or anticipated, potentially doubling or tripling market size. The global incidence of BPDCN, although low (estimated at fewer than 1 in 10 million annually), makes international sales vital for long-term growth.
Pipeline and Off-Label Uses: Investigations into other CD123-positive malignancies have the potential to diversify revenue streams. If clinical trials demonstrate positive outcomes, ELZONRIS could be approved for additional indications, broadening its market and elevating revenue projections.

Forecasts and Revenue Projections
Industry analysts project that ELZONRIS could reach peak sales of $200-300 million annually within a decade, primarily driven by international expansion and potential label expansions. The growth rate remains moderate, constrained by disease rarity but bolstered by increasing clinical acceptance and pipeline activity. Such forecasts assume steady reimbursement policies and continued clinical success.

Profitability and Cost Considerations
The biologic manufacturing process for ELZONRIS involves complex cell culture systems and lyophilization, leading to high production costs. These expenses are balanced against premium pricing and limited patient population, enabling margins to remain favorable. R&D costs for pipeline extensions and post-marketing studies further influence long-term profitability.

Competitive Landscape and Future Outlook

While ELZONRIS remains the front-line targeted therapy for BPDCN, emerging treatments threaten to encroach on its market share. Notably, efforts to develop CD123-directed bispecific antibodies and CAR-T therapies have gained momentum:

Bispecific Antibodies: Agents like flotetuzumab and others in early clinical phases aim to harness T-cell engagement, potentially offering less toxicity and oral options.
CAR-T Therapies: Customized cellular therapies targeting CD123 are under clinical evaluation and could revolutionize treatment protocols if safety and efficacy are established.

Nonetheless, ELZONRIS’s proven efficacy, regulatory backing, and existing manufacturing infrastructure position it favorably, provided clinical development of competitors encounters setbacks.

Regulatory and Market Access Risks

Pricing and Reimbursement: Stringent payer policies and the push for value-based care could threaten high price margins, especially outside the U.S.
Patent and Exclusivity Limitations: The expiration of orphan exclusivity in 2028 raises the possibility of biosimilar competition, though the complex biologic manufacturing process and patent protections can delay entry.
Pipeline Setbacks: Unfavorable trial results or safety concerns could reduce the drug’s attractiveness, impacting revenue forecasts.

Key Takeaways

Market Position: ELZONRIS’s significant role as a first-in-class biologic for BPDCN secures its niche despite the small patient population.
Growth Potential: International expansion and pipeline development—particularly into other CD123-positive diseases—are pivotal to augmenting revenues.
Financial Outlook: Estimated peak annual revenues of $200-300 million are contingent upon successful market penetration and clinical development milestones.
Competitive Risks: Emerging immunotherapies pose future threat, but clinical validation delays or setbacks could preserve ELZONRIS’s market dominance.
Strategic Considerations: Maintaining robust manufacturing, navigating patent protections, and securing reimbursement are critical to optimizing ELZONRIS’s financial trajectory.

FAQs

What therapeutic niche does ELZONRIS occupy in oncology?
ELZONRIS is a targeted biologic therapy approved for BPDCN, a rare hematologic cancer, representing the first specific immunotherapy approved for this indication.
How does ELZONRIS compare commercially to other biologic cancer therapies?
Its revenues are modest compared to blockbuster biologics due to the ultra-rare nature of BPDCN but are significant within the orphan drug segment, justified by high per-treatment pricing and limited competition.
What are the prospects for ELZONRIS’s global expansion?
Regulatory approvals are underway in Europe and Asia, which could substantially increase its market if aligned with reimbursement frameworks.
What emerging therapies could impact ELZONRIS’s market share?
CD123-targeted bispecific antibodies and CAR-T cell therapies are in clinical development and may challenge ELZONRIS’s positioning if they demonstrate superior safety and efficacy.
When is ELZONRIS expected to face biosimilar competition?
Biosimilar entries are unlikely before 2028, coinciding with the expiration of orphan exclusivity, after which patent protections and manufacturing complexities may delay immediate biosimilar access.

References

[1] FDA. ELZONRIS (tagraxofusp-erzs) approval announcement. 2018.
[2] Stemline Therapeutics. ELZONRIS prescribing information. 2022.
[3] Market research reports on hematologic malignancies and biologic drug sales forecasts.
[4] Clinical trial registries and recent publications on CD123-directed therapies.
[5] Moderna's acquisition details and corporate disclosures.

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