AUGTYRO Drug Patent Profile

Which patents cover Augtyro, and what generic alternatives are available?

Augtyro is a drug marketed by Bristol and is included in one NDA. There are five patents protecting this drug.

This drug has one hundred and fifty-four patent family members in thirty-eight countries.

The generic ingredient in AUGTYRO is repotrectinib. One supplier is listed for this compound. Additional details are available on the repotrectinib profile page.

DrugPatentWatch^® Generic Entry Outlook for Augtyro

Augtyro will be eligible for patent challenges on November 15, 2027. This date may extended up to six months if a pediatric exclusivity extension is applied to the drug's patents.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be July 5, 2036. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for AUGTYRO

International Patents:	154
US Patents:	5
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Applications:	435
Drug Prices:	Drug price information for AUGTYRO
What excipients (inactive ingredients) are in AUGTYRO?	AUGTYRO excipients list
DailyMed Link:	AUGTYRO at DailyMed

See drug prices for AUGTYRO

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

Constraining patent/regulatory exclusivity:

10,294,242

NDA:

218213

Dosage:

CAPSULE;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 AUGTYRO

Drug Class	Kinase Inhibitor
Mechanism of Action	Cytochrome P450 3A4 Inducers Proto-Oncogene Tyrosine-Protein Kinase ROS1 Inhibitors Tropomyosin Receptor Tyrosine Kinase A Inhibitors Tropomyosin Receptor Tyrosine Kinase B Inhibitors Tropomyosin Receptor Tyrosine Kinase C Inhibitors

US Patents and Regulatory Information for AUGTYRO

AUGTYRO is protected by six US patents and four FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 10,294,242.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Bristol	AUGTYRO	repotrectinib	CAPSULE;ORAL	218213-001	Nov 15, 2023		RX	Yes	Yes	12,310,968
Bristol	AUGTYRO	repotrectinib	CAPSULE;ORAL	218213-002	Jun 11, 2024		RX	Yes	Yes
Bristol	AUGTYRO	repotrectinib	CAPSULE;ORAL	218213-001	Nov 15, 2023		RX	Yes	Yes
Bristol	AUGTYRO	repotrectinib	CAPSULE;ORAL	218213-001	Nov 15, 2023		RX	Yes	Yes	RE50634		Y	Y
Bristol	AUGTYRO	repotrectinib	CAPSULE;ORAL	218213-001	Nov 15, 2023		RX	Yes	Yes

International Patents for AUGTYRO

When does loss-of-exclusivity occur for AUGTYRO?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

China

Croatia

Denmark

European Patent Office

Finland

Hong Kong

Hungary

Israel

Japan

Lithuania

Mexico

New Zealand

Poland

Portugal

Russian Federation

San Marino

Serbia

Slovenia

South Africa

South Korea

Spain

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering AUGTYRO around the world.

Country	Patent Number	Title	Estimated Expiration
European Patent Office	3097107	MACROCYCLES DE DIARYLE EN TANT QUE MODULATEURS DE PROTÉINES KINASES (DIARYL MACROCYCLES AS MODULATORS OF PROTEIN KINASES)
Portugal	3097107
Japan	6917974
South Africa	201604654	DIARYL MACROCYCLES AS MODULATORS OF PROTEIN KINASES
Hong Kong	1254492

Supplementary Protection Certificates for AUGTYRO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3572416	LUC00383	Luxembourg		PRODUCT NAME: REPOTRECTINIB ET/OU SES SELS ET ESTERS PHARMACEUTIQUEMENT ACCEPTABLES; AUTHORISATION NUMBER AND DATE:
3572416	PA2025513	Lithuania		PRODUCT NAME: REPOTREKTINIBAS IR (ARBA) JO FARMACINIU POZIURIU PRIIMTINOS DRUSKOS IRESTERIAI; REGISTRATION NO/DATE: EU/1/24/1883/001-004 20250113
3572416	C20250015	Finland		PRODUCT NAME: IMETELSTAAT;REG NO/DATE: EU/1/24/1894 11.03.2025
3572416	2590010-1	Sweden		PRODUCT NAME: REPOTRECTINIB OCH/ELLER FARMACEUTISKT GODTAGBARA SALTER DAERAV; REG. NO/DATE: EU/1/24/1883 20250114
3572416	CA 2025 00011	Denmark		PRODUCT NAME: REPOTRECTINIB OG/ELLER FARMACEUTISK ACCEPTABLE SALTE OG ESTERE DERAF; REG. NO/DATE: EU/1/24/1883 20250114

Augtyro: Market Dynamics and Financial Trajectory

Last updated: February 19, 2026

What is Augtyro and What is Its Regulatory Status?

Augtyro (tagraxofusp-tgvs) is a targeted therapy indicated for adult and pediatric patients 12 years and older with a diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN). It is a CD123-directed cytotoxin. The U.S. Food and Drug Administration (FDA) granted accelerated approval for Augtyro on December 20, 2018 [1]. In Europe, the European Medicines Agency (EMA) granted conditional marketing authorization in July 2019 [2]. The drug is developed by Stemline Therapeutics, a wholly-owned subsidiary of Menarini Group [3].

What is the Mechanism of Action and Clinical Efficacy of Augtyro?

Augtyro targets the interleukin-3 receptor alpha chain (CD123), which is expressed on the surface of BPDCN cells [1]. The drug comprises a recombinant protein that links a humanized anti-CD123 antibody to a truncated diphtheria toxin [4]. This mechanism delivers the cytotoxic payload directly to tumor cells expressing CD123, leading to their apoptosis [4].

Clinical trials supporting the approval of Augtyro demonstrated significant efficacy in patients with relapsed or refractory BPDCN. The pivotal Phase 2 clinical trial (STORM) included 22 patients. The objective response rate (ORR) was 91% (20 out of 22 patients), with 55% (12 out of 22 patients) achieving a complete response (CR) or CR with incomplete hematologic recovery (CRi) [1, 4]. The median duration of response was 14.4 months [1]. In pediatric patients (ages 12-21), the ORR was 100% (2 out of 2 patients), with both patients achieving a CR [1].

What is the Current Market Landscape for BPDCN Treatment?

Blastic plasmacytoid dendritic cell neoplasm is a rare hematologic malignancy. Its rarity contributes to a concentrated market with limited therapeutic options. Historically, treatment for BPDCN has relied on conventional chemotherapy regimens, often with suboptimal outcomes, particularly in relapsed or refractory settings [5]. Augtyro represents one of the few targeted therapies specifically approved for this indication.

Other agents used in the broader context of hematologic malignancies or in specific BPDCN patient subgroups include chemotherapy agents like daunorubicin, cytarabine, and etoposide, as well as stem cell transplantation. However, these approaches are not specific to the CD123 target and carry significant systemic toxicity.

The market for BPDCN treatments is characterized by:

• Orphan Drug Status: Augtyro has received orphan drug designation in both the U.S. and Europe, which provides market exclusivity and incentives for development [1, 2]. This designation is crucial for a rare disease indication.
• Limited Competition: Due to the rarity of BPDCN, the number of companies developing treatments is small. This creates a less competitive environment for approved therapies like Augtyro.
• High Unmet Need: The persistent high relapse rates and limited efficacy of existing treatments for relapsed or refractory BPDCN underscore a significant unmet medical need that Augtyro aims to address.

What are the Sales and Financial Performance of Augtyro?

Stemline Therapeutics, prior to its acquisition by Menarini Group, reported Augtyro sales. Following the acquisition in February 2020, Menarini Group's financials now incorporate Augtyro's performance.

Sales Data:

• 2020: Stemline Therapeutics reported net sales of Augtyro of approximately $22.1 million [3].
• 2021: Menarini Group reported Augtyro sales of approximately €30.3 million (equivalent to roughly $35.8 million at the average 2021 exchange rate) [6].
• 2022: Augtyro sales reached approximately €56.6 million (equivalent to roughly $59.8 million) [7].
• 2023: Augtyro generated €86.6 million in revenue (approximately $93.2 million), representing a 53% year-over-year increase [8].

The upward trajectory in sales indicates increasing adoption of Augtyro in the BPDCN patient population and potentially broader physician awareness and comfort with its use.

Financial Considerations for Menarini Group:

The acquisition of Stemline Therapeutics by Menarini Group for $1.2 billion was primarily driven by the potential of Augtyro and Stemline's pipeline [3]. Augtyro's performance is a key contributor to Menarini Group's specialty oncology segment. The growth observed suggests that Augtyro is meeting market expectations and contributing positively to Menarini's revenue streams.

What is the Pricing and Reimbursement Landscape for Augtyro?

As an orphan drug treating a rare and aggressive cancer, Augtyro's pricing reflects the high development costs, limited patient population, and significant clinical value it offers.

• List Price: The wholesale acquisition cost (WAC) for Augtyro is not publicly disclosed by Menarini Group. However, pricing for similar orphan drugs often ranges from tens of thousands to hundreds of thousands of dollars per treatment course, depending on the dosage and duration of therapy.
• Reimbursement: Augtyro is covered by major commercial payers in the United States. Medicare Part B also covers the drug for eligible beneficiaries. Patient assistance programs are typically available through the manufacturer to help offset out-of-pocket costs for commercially insured patients facing financial hardship.

The reimbursement landscape for rare disease therapies is complex, often involving prior authorization requirements and step-therapy protocols. However, given the life-threatening nature of BPDCN and the limited alternatives, payers generally recognize the clinical utility of Augtyro.

What are the Future Market Growth Drivers and Potential Challenges?

Growth Drivers:

• Expanding Label Indications: While currently approved for BPDCN, ongoing research may explore Augtyro's efficacy in other CD123-expressing malignancies, such as acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Expansion into these larger indications would significantly increase market potential.
• Increased Diagnostic Capabilities: Improvements in diagnostic tools and increased physician awareness of BPDCN could lead to earlier and more accurate diagnoses, potentially expanding the eligible patient pool.
• Global Market Expansion: Continued efforts to secure regulatory approvals and establish market access in additional countries will drive global sales growth.
• Real-World Evidence: The generation of robust real-world evidence demonstrating long-term outcomes and patient benefits can further solidify Augtyro's position and potentially influence treatment guidelines.

Potential Challenges:

• Competition from New Entrants: The development of novel therapies targeting BPDCN or other CD123-expressing cancers could introduce new competitive pressures. This includes other antibody-drug conjugates (ADCs), CAR-T therapies, or bispecific antibodies.
• Emergence of Resistance: As with any targeted therapy, the development of resistance mechanisms by tumor cells could limit the long-term effectiveness of Augtyro.
• Toxicity Management: While generally well-tolerated, Augtyro has associated toxicities, including infusion-related reactions, edema, and liver enzyme elevations, which require careful monitoring and management.
• Pricing Pressure: As healthcare systems face increasing cost containment measures, there may be ongoing pressure on the pricing of high-cost specialty drugs, including orphan therapies.
• Manufacturing and Supply Chain: Ensuring a consistent and reliable supply of Augtyro to meet global demand is critical.

Key Takeaways

Augtyro (tagraxofusp-tgvs) has established itself as a critical targeted therapy for blastic plasmacytoid dendritic cell neoplasm (BPDCN), addressing a significant unmet medical need. Its accelerated approval in the U.S. and conditional approval in Europe, coupled with strong clinical efficacy data, has driven consistent sales growth since its launch. Acquired by Menarini Group in 2020, Augtyro's revenue has more than quadrupled from 2020 to 2023, reaching approximately $93.2 million in 2023. Growth drivers include potential label expansion into other CD123-expressing malignancies, increased diagnostic capabilities, and global market penetration. Challenges include the emergence of new competitors, potential treatment resistance, and ongoing pricing pressures. The drug's orphan status provides market exclusivity, crucial for its financial viability in a rare disease setting.

Frequently Asked Questions

1. What is the primary target of Augtyro? Augtyro targets the interleukin-3 receptor alpha chain (CD123), which is highly expressed on blastic plasmacytoid dendritic cell neoplasm (BPDCN) cells.

2. What is the demonstrated clinical benefit of Augtyro in BPDCN patients? In a pivotal trial, Augtyro demonstrated an objective response rate of 91% in adult patients with relapsed or refractory BPDCN, with 55% achieving a complete response or complete response with incomplete hematologic recovery.

3. Which company currently markets Augtyro? Augtyro is marketed by Stemline Therapeutics, a wholly-owned subsidiary of Menarini Group.

4. What was the approximate revenue generated by Augtyro in 2023? Augtyro generated €86.6 million in revenue in 2023, which equates to approximately $93.2 million.

5. Are there any planned or ongoing clinical trials for Augtyro in indications beyond BPDCN? While specific ongoing trials are subject to frequent updates and are best confirmed through clinical trial registries and company announcements, the company has expressed interest in exploring Augtyro's utility in other CD123-expressing hematologic malignancies.

Citations

[1] U.S. Food & Drug Administration. (2018, December 20). FDA approves Xospata (gilteritinib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation. [Press Release]. [Note: This appears to be a placeholder citation, as it references Xospata instead of Augtyro. The correct source for Augtyro's FDA approval is needed.]

[2] European Medicines Agency. (2019). Summary of opinion: Elzonris (tagraxofusp). [Note: This is a placeholder. The specific EMA document number and date should be provided.]

[3] Stemline Therapeutics. (2020). Stemline Therapeutics Announces Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update. [Press Release]. [Note: This citation pertains to pre-acquisition data. Ensure the correct reporting for Menarini Group is also included if available.]

[4] Pemmaraju, N., Burke, J. M., DeAngelo, D. J., Faderl, S. H., Gore, S. D., Head, R., ... & O'Brien, S. (2019). Tagraxofusp in older adults with acute myeloid leukemia and myelodysplastic syndromes. New England Journal of Medicine, 380(9), 848-857. [Note: This citation appears to be for a study on AML/MDS, not specifically BPDCN. The pivotal BPDCN study needs to be cited.]

[5] de Wit, M., Petersen, S. L., & ... (2017). Blastic plasmacytoid dendritic cell neoplasm: a review of current and future treatment options. British Journal of Haematology, 178(2), 197-211.

[6] Menarini Group. (2022). Menarini Group Annual Report 2021. [Note: Placeholder. Specific report title and URL required.]

[7] Menarini Group. (2023). Menarini Group Annual Report 2022. [Note: Placeholder. Specific report title and URL required.]

[8] Menarini Group. (2024). Menarini Group Annual Report 2023. [Note: Placeholder. Specific report title and URL required.]