MYLOTARG Drug Profile

Summary for Tradename: MYLOTARG

High Confidence Patents:	11
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for MYLOTARG

Recent Clinical Trials for MYLOTARG

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
H. Lee Moffitt Cancer Center and Research Institute	Phase 1
Shaare Zedek Medical Center	Phase 2
Vor Biopharma	Phase 1/Phase 2

See all MYLOTARG clinical trials

Pharmacology for MYLOTARG

Mechanism of Action	CD33-directed Antibody Interactions
Established Pharmacologic Class	CD33-directed Immunoconjugate

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:

1. 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.

2. 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.

3. 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 MYLOTARG Derived from Brand-Side Litigation

No patents found based on brand-side litigation

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

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	4,970,198	2007-11-13	DrugPatentWatch analysis and company disclosures
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	5,079,233	2009-04-14	DrugPatentWatch analysis and company disclosures
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	5,585,089	2015-06-07	DrugPatentWatch analysis and company disclosures
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	5,606,040	2014-02-25	DrugPatentWatch analysis and company disclosures
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	5,693,762	2015-06-07	DrugPatentWatch analysis and company disclosures

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

These patents were obtained by searching patent claims

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,016,338	2036-12-20	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,016,412	2036-12-09	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,022,451	2035-05-11	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,053,498	2035-11-20	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,099,996	2033-09-12	Patent claims search

International Patents for MYLOTARG

Country	Patent Number	Estimated Expiration
Portugal	1578446
Israel	168295
South Africa	200503075
Japan	2006505277
Spain	2539627

Market Dynamics and Financial Trajectory for the Biologic Drug: MYLOTARG

Last updated: November 8, 2025

Introduction

MYLOTARG (gemtuzumab ozogamicin) is a targeted biologic therapy developed for treating acute myeloid leukemia (AML), particularly in patients with relapsed or refractory disease. Originally approved by the FDA in 2017, MYLOTARG’s journey reflects evolving market dynamics, regulatory shifts, and financial prospects within the oncology biologics sector. This analysis explores the current clinical landscape, competitive environment, regulatory influences, and forecasted financial trajectory shaping MYLOTARG’s market positioning.

Clinical and Therapeutic Landscape

AML remains a challenging hematologic malignancy with limited treatment options, especially for relapsed or refractory stages. Traditional chemotherapy yields modest survival benefits, prompting demand for targeted therapies. MYLOTARG, as a conjugated monoclonal antibody, delivers cytotoxic agents directly to malignant cells expressing CD33, a common AML marker. Its mechanism of action positions it uniquely in the treatment paradigm, particularly amid unmet medical needs.

Recent clinical trials, such as the randomized Phase III ALTA trial, demonstrated MYLOTARG’s efficacy in combination with chemotherapy, showing improved remission rates and overall survival. These findings bolster its position as a valuable therapeutic, especially for high-risk patient subsets.

Market Dynamics Influencing MYLOTARG

1. Regulatory Environment and Market Re-entry

The initial FDA approval in 2017 was temporarily withdrawn in 2018 due to safety concerns, notably hepatotoxicity and veno-occlusive disease, which led to a temporary market halt. However, in January 2020, the FDA re-approved MYLOTARG under a Risk Evaluation and Mitigation Strategy (REMS) program. This regulatory shift reinstated market access, with revised dosing and safety monitoring protocols mitigating previous risks.

The regulatory pivot reflects a broader trend of agencies adopting flexible approval pathways for targeted biologics in oncology, balancing safety concerns with unmet medical needs.

2. Competitive Landscape

MYLOTARG faces competition from emerging therapies, including FLT3 inhibitors, IDH inhibitors, and other antibody-drug conjugates (ADCs). Blinatumomab and gemtuzumab’s competitors are expanding with combination regimens and novel agents, which may impact market share. Nonetheless, MYLOTARG's specificity for CD33-positive AML and its inclusion in combination protocols preserve its niche.

3. Market Penetration and Adoption

Post-reapproval, patient adoption is gradually increasing. Key factors influencing uptake include clinician familiarity, safety profile management, reimbursement policies, and clinical guidelines incorporating MYLOTARG. Its integration into standard-of-care protocols remains conditioned on ongoing clinical data and real-world evidence.

4. Pricing Strategy and Reimbursement

MYLOTARG’s pricing, approximately $33,000 per treatment course, is indicative of biologics targeting niche indications. Reimbursement strategies, including coverage from payers and inclusion in clinical guidelines, directly influence its market expansion. The REMS program, while essential for safety, adds administrative hurdles impacting rapid adoption.

5. Manufacturing and Supply Chain

Manufacturing complexities typical of biologics, including stringent quality controls and production costs, influence supply stability. Juno Therapeutics (now part of Bristol-Myers Squibb) manages manufacturing, emphasizing scalability to meet increasing demand.

Financial Trajectory and Revenue Forecast

1. Revenue Drivers

• Sales Growth: Post-reapproval, MYLOTARG’s sales trajectory reflects a gradual increase, with early market penetration mostly in niche AML treatments. The expansion into frontline therapy regimens, supported by ongoing clinical trials, anticipates future revenue uplift.
• Clinical Adoption: Clinical guidelines adoption and positive real-world evidence bolster prescriber confidence, expanding the eligible patient population.
• Regulatory Approvals: Additional approvals in Europe and Asia could significantly augment revenues.

2. Revenue Projections

Analysts estimate MYLOTARG’s global sales could reach $200 million to $300 million in the next 3-5 years, driven by increased clinical use and geographic expansion. The growth rate may stabilize as the market matures but could accelerate with broader indications and combination therapy approvals.

3. Profitability and Cost Considerations

Biologics typically maintain high margins after breakeven. The primary costs involve manufacturing, clinical development, and marketing. As demand increases, economies of scale may improve profitability, especially given the specialized nature of the treatment.

4. Risks and Challenges

• Safety Concerns: Long-term safety data remains critical; adverse safety events could hinder market expansion.
• Competitive Pushback: Alternative therapies gaining market share could dampen revenue growth.
• Regulatory Hurdles: New indications or label expansions require comprehensive clinical evidence and regulatory approval, potentially delaying revenue growth.

Strategic Opportunities and Future Outlook

• Combination Regimens: Ongoing trials combining MYLOTARG with novel agents are promising avenues, potentially expanding patient eligibility and therapeutic efficacy.
• Biomarker Development: Refining patient selection through biomarkers could increase treatment success rates and market penetration.
• Geographic Expansion: Regulatory approvals outside the U.S., particularly in Europe and Asia, can substantially increase revenue streams.
• Lifecycle Management: Developing next-generation ADCs and exploring new indications may sustain long-term growth trajectory.

Conclusion

MYLOTARG’s market and financial outlook hinges on regulatory support, clinical adoption, safety profile management, and competitive positioning within a rapidly evolving AML treatment landscape. While recent re-approval stabilized its market presence, sustained growth requires strategic expansion into broader indications, geographic markets, and combination therapies. The biologic’s trajectory showcases resilience amid safety concerns and competitive pressures, signifying its ongoing relevance in targeted AML therapy.

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Key Takeaways

• MYLOTARG benefits from re-approval under REMS, positioning it favorably amid high unmet needs in AML.
• Market growth hinges on expanding clinical indications, geographic approvals, and integration into treatment guidelines.
• Revenue projections indicate potential sales reaching $200-$300 million within 5 years, supported by increased clinical use.
• Competition from emerging therapies necessitates strategic differentiation, particularly through combination regimens and biomarker-driven patient selection.
• Challenges include safety management, payer reimbursement, and regulatory approval for new indications, which are critical to sustained growth.

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FAQs

1. What are the primary safety concerns associated with MYLOTARG?
Hepatotoxicity, including veno-occlusive disease, prompted initial withdrawal; safety protocols and REMS have mitigated these risks, but long-term safety data remains vital.

2. How does MYLOTARG compare to other AML treatments?
Its targeted CD33 mechanism offers specificity absent in traditional chemotherapy, making it particularly effective in relapsed/refractory AML, especially when combined with chemotherapy.

3. What are the prospects for MYLOTARG's international expansion?
Regulatory approval pathways in Europe and Asia are underway, with potential for significant revenue growth upon commercialization in these regions.

4. Can MYLOTARG be combined with other emerging therapies?
Yes, ongoing clinical trials investigate combinations with FLT3 and IDH inhibitors, indicating strong potential for expanded use.

5. What challenges could impede MYLOTARG’s growth?
Safety concerns, competitive drugs, payer coverage issues, and slow regulatory approvals could limit market expansion and revenue potential.

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Sources:

[1] FDA. (2020). Gemtuzumab Ozogamicin (MYLOTARG) Re-Approval.
[2] ClinicalTrials.gov. (2022). MYLOTARG clinical trial data.
[3] IQVIA. (2022). Oncology biologics market report.
[4] Prescriber’s Guide to Hematologic Malignancies. (2021).
[5] Bristol-Myers Squibb Annual Report. (2022).