MYLOTARG Drug Profile

Summary for Tradename: MYLOTARG

High Confidence Patents:	0
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for MYLOTARG
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases 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

Recent Litigation for MYLOTARG

Identify key patents and potential future biosimilar entrants

District Court Litigation

Case Name	Date
AbbVie Inc. v. Alvotech hf.	2021-05-28
Alvotech USA Inc. v. Abbvie Inc	2021-05-11
AbbVie Inc. v. Alvotech hf.	2021-04-27

See all MYLOTARG litigation

PTAB Litigation

Petitioner	Date
	2017-12-20

See all MYLOTARG litigation

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. General brand-side disclosures

These patents were identified from searching 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 Company Disclosures

No patents found based on 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	2033-03-11	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,016,412	2021-05-01	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,022,451	2039-02-26	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,053,498	2034-11-21	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,099,996	2032-01-18	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,111,968	2032-02-15	Patent claims search
Wyeth Pharmaceuticals Llc	MYLOTARG	gemtuzumab ozogamicin	For Injection	761060	10,117,943	2039-02-26	Patent claims search

Market Dynamics and Financial Trajectory for the Biologic Drug: Mylotarg

Introduction

Mylotarg, also known as gemtuzumab ozogamicin, is a biologic drug that has had a complex and tumultuous history in the treatment of acute myeloid leukemia (AML). This article delves into the market dynamics and financial trajectory of Mylotarg, highlighting its approvals, withdrawals, and current market status.

Initial Approval and Early Market Presence

Mylotarg was first approved by the FDA in May 2000 under the accelerated approval program for the treatment of patients aged 60 and older with recurrent AML who were not candidates for other chemotherapy. This approval was based on a surrogate endpoint of response rate observed in clinical trials[3][4].

Voluntary Withdrawal from the Market

In 2010, Pfizer voluntarily withdrew Mylotarg from the U.S. market at the request of the FDA. This decision was made after a confirmatory clinical trial failed to show any improvement in clinical benefit, and there was a higher incidence of patient deaths in the Mylotarg arm of the study due to treatment-related toxicities. The drug was associated with serious liver conditions, including veno-occlusive disease, which could be fatal[3][4].

Reapproval and Second Market Entry

After several years, Mylotarg was reapproved by the FDA in 2017 for the treatment of adults with newly diagnosed AML whose tumors express the CD33 antigen, as well as for treatment in the second-line setting for patients over two years old with CD33-positive AML. This reapproval was based on new Phase 3 studies, including the ALFA-0701 study, which showed better results for patients receiving Mylotarg plus chemotherapy compared to those on chemotherapy alone[4].

Market Size and Forecast

The global market for Mylotarg was estimated to be worth US$ 214.3 million in 2023. However, it is forecast to decrease to a readjusted size of US$ 179.5 million by 2030, with a compound annual growth rate (CAGR) of -2.5% during the forecast period of 2024-2030. This decline reflects the challenges and uncertainties associated with the drug's history and its niche market position[1].

Pricing and Cost Considerations

Upon its reapproval, Pfizer priced Mylotarg at $24,600 for one cycle of treatment, which is higher than its list price of $15,522 when it was initially withdrawn. Despite the higher price, Pfizer noted that few patients would have out-of-pocket costs, as the drug is priced below other recently approved AML treatments[4].

Safety and Efficacy Concerns

Mylotarg's label includes a boxed warning for hepatotoxicity, including severe or fatal hepatic veno-occlusive disease (VOD), which has been observed in patients taking the drug alone and in combination therapy. The drug's safety profile has been a significant factor in its market dynamics, influencing both its initial withdrawal and its current use[3][4].

Competitive Landscape

The AML treatment market is competitive, with several other drugs and therapies available. Mylotarg's unique mechanism as an antibody drug conjugate (ADC) allows it to target cancer cells more precisely, but it must compete with other targeted therapies and traditional chemotherapies. The competitive landscape is further complicated by the drug's history of safety concerns and the need for careful patient selection[4].

Patient Population and Indications

Mylotarg is currently approved for a specific patient population: adults with newly diagnosed AML whose tumors express the CD33 antigen, and for second-line treatment in patients over two years old with CD33-positive AML. This targeted approach helps in managing the drug's risks and benefits more effectively[4].

Financial Impact on Pfizer

The financial impact of Mylotarg on Pfizer is significant, given the drug's complex history and the costs associated with its development, clinical trials, and marketing. While the drug generates revenue, its market size and growth are limited by its niche indications and safety concerns. Pfizer's overall financial performance is influenced by a diverse portfolio of drugs, but Mylotarg remains an important, albeit challenging, part of its oncology offerings[1][3].

Regulatory Environment

The regulatory environment plays a crucial role in the market dynamics of Mylotarg. The FDA's accelerated approval program allowed the drug to reach the market quickly, but it also required Pfizer to conduct additional clinical trials to confirm the drug's benefit. The FDA's strict oversight and the requirement for post-marketing studies have significantly impacted Mylotarg's market trajectory[3][4].

Future Outlook

The future outlook for Mylotarg is cautious, given its history and the ongoing need to manage its safety profile. The drug's market size is expected to decline, but it remains a valuable treatment option for a specific subset of AML patients. Continued monitoring and research will be essential to optimize its use and mitigate its risks.

Key Takeaways

• Market Size and Forecast: The global market for Mylotarg is forecast to decrease to US$ 179.5 million by 2030.
• Safety Concerns: Mylotarg is associated with serious liver conditions, including veno-occlusive disease.
• Reapproval: The drug was reapproved in 2017 with a new dosing regimen and for a different patient population.
• Pricing: Mylotarg is priced at $24,600 per cycle, higher than its initial list price.
• Competitive Landscape: The drug competes in a competitive AML treatment market with other targeted therapies.
• Regulatory Environment: The FDA's accelerated approval program and post-marketing studies have significantly impacted Mylotarg's market trajectory.

FAQs

What is Mylotarg used for?

Mylotarg is used for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) whose tumors express the CD33 antigen, as well as for second-line treatment in patients over two years old with CD33-positive AML[4].

Why was Mylotarg withdrawn from the market in 2010?

Mylotarg was withdrawn from the market in 2010 due to a confirmatory clinical trial that failed to show any improvement in clinical benefit and a higher incidence of patient deaths due to treatment-related toxicities[3][4].

What is the current market size and forecast for Mylotarg?

The global market for Mylotarg was estimated to be worth US$ 214.3 million in 2023 and is forecast to decrease to US$ 179.5 million by 2030 with a CAGR of -2.5% during the forecast period of 2024-2030[1].

What are the safety concerns associated with Mylotarg?

Mylotarg is associated with serious liver conditions, including severe or fatal hepatic veno-occlusive disease (VOD)[3][4].

How much does Mylotarg cost?

Mylotarg is priced at $24,600 for one cycle of treatment, which is higher than its list price when it was initially withdrawn[4].

Sources

1. Valuates Reports: Global Mylotarg Industry Research Report.
2. Vor Bio: Second Quarter 2024 Financial Results and Provides Business Update.
3. PR Newswire: FDA: Pfizer Voluntarily Withdraws Cancer Treatment Mylotarg From U.S. Market.
4. BioPharma Dive: Pfizer's Mylotarg gets a second shot at market.