Ravulizumab-cwvz - Biologic Drug Details

Summary for ravulizumab-cwvz

Tradenames:	1
High Confidence Patents:	9
Applicants:	1
BLAs:	1
Suppliers: see list	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ravulizumab-cwvz

Recent Litigation for ravulizumab-cwvz

Identify key patents and potential future biosimilar entrants

District Court Litigation

Case Name	Date
Chugai Pharmaceutical Co., Ltd. v. Alexion Pharmaceuticals, Inc.	2018-11-15
Oasis Research, LLC v. Adrive, LLC	2010-08-30
Energy Transportation Group Inc. v. Sonic Innovations Inc.	2005-06-23

See all ravulizumab-cwvz litigation

Pharmacology for ravulizumab-cwvz

Mechanism of Action	Complement Inhibitors
Established Pharmacologic Class	Complement Inhibitor

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 ravulizumab-cwvz Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for ravulizumab-cwvz Derived from Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	10,227,400	2034-03-07	Company disclosures
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	10,584,164	2039-01-14	Company disclosures
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	11,365,241	2038-07-27	Company disclosures
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	9,079,949	2034-03-07	Company disclosures
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	9,107,861	2034-03-07	Company disclosures
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	9,206,251	2034-03-07	Company disclosures
Alexion Pharmaceuticals, Inc.	ULTOMIRIS	ravulizumab-cwvz	Injection	761108	9,371,377	2034-03-07	Company disclosures

3) Low Certainty: US Patents for ravulizumab-cwvz 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

International Patents for ravulizumab-cwvz

Country	Patent Number	Estimated Expiration
World Intellectual Property Organization (WIPO)	2015134894
Montenegro	02629
Israel	273337
Japan	2021036870
China	106459189
Australia	2019236617
Taiwan	201542590

Supplementary Protection Certificates for ravulizumab-cwvz

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
C03095795/01	Switzerland		PRODUCT NAME: RAVULIZUMAB; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67278 16.02.2023
C02970455/01	Switzerland		PRODUCT NAME: RAVULIZUMAB; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67278 24.08.2021
C03095795/02	Switzerland		PRODUCT NAME: RAVULIZUMAB; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67278 29.08.2023

Market Dynamics and Financial Trajectory for Ravulizumab-cwvz (ULTOMIRIS)

Introduction

Ravulizumab-cwvz, marketed as ULTOMIRIS, is a groundbreaking biologic drug developed by Alexion Pharmaceuticals, Inc. for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other rare diseases. This article delves into the market dynamics and financial trajectory of ULTOMIRIS, highlighting its approval, efficacy, cost-effectiveness, and the company's strategic plans.

FDA Approval and Clinical Efficacy

On December 21, 2018, the FDA approved ULTOMIRIS for adult patients with PNH, based on two pivotal phase 3 studies: ALXN1210-PNH-301 and ALXN1210-PNH-302. These studies demonstrated the non-inferiority of ULTOMIRIS compared to eculizumab, another C5 complement inhibitor, in terms of transfusion avoidance, reduction of hemolysis, and stabilization of hemoglobin levels[1][5].

Market Positioning

ULTOMIRIS is the first and only long-acting C5 complement inhibitor administered every eight weeks, offering a significant advantage over eculizumab, which requires dosing every two weeks. This extended dosing interval can improve patient compliance and reduce the burden on healthcare systems[5].

Cost-Effectiveness

While the loading dose of ULTOMIRIS is more expensive than eculizumab, the maintenance doses are less costly. Over a lifetime treatment horizon, ULTOMIRIS is potentially less costly and equally effective compared to eculizumab. However, the cost savings are realized only after several years of treatment, with treatment-naive patients needing to receive ULTOMIRIS for more than 26 years to achieve cost savings[3].

Financial Performance and Growth

Alexion Pharmaceuticals has executed a strong financial and operational performance, underscored by significant revenue and non-GAAP EPS growth. In Q3 2019, the company reported 23% revenue growth and 38% non-GAAP EPS growth year-over-year. The company's strategy to drive growth includes facilitated conversion to ULTOMIRIS, expansion into new diseases, and diversification of the portfolio[2].

Strategic Expansion

Alexion has redefined its strategy to focus on driving conversion to ULTOMIRIS, expanding current assets into new diseases such as neurology, and diversifying its portfolio. The company has rebuilt and advanced its pipeline, including the delivery of three positive Phase 3 programs and the initiation of multiple additional clinical programs. This includes positive topline results from a Phase 3 study of ULTOMIRIS in adults with generalized myasthenia gravis (gMG), which further expands the drug's potential market[2][4].

Pipeline and Future Launches

Alexion's pipeline includes 22 clinical-stage development programs across 11 assets, with the potential to deliver at least 10 additional launches by 2023. This robust pipeline, supported by a revamped and strengthened R&D organization and disciplined business development initiatives, positions the company for sustained growth and value creation[2].

Regulatory and Commercial Plans

ULTOMIRIS has received regulatory approvals in the U.S. and is under review in the European Union and Japan for the treatment of PNH. The company's future clinical, regulatory, and commercial plans for ULTOMIRIS and other products are focused on maintaining a strong culture of compliance and transparency while driving growth and durability[2][5].

Safety and Tolerability

ULTOMIRIS has been well tolerated in clinical studies, with a safety profile consistent with that observed in Phase 3 studies for PNH and atypical hemolytic uremic syndrome (aHUS). The most frequent adverse reactions include upper respiratory infection and headache[1][4].

Economic Impact

The economic impact of ULTOMIRIS is significant, given its potential to reduce healthcare costs over the long term. However, the high initial costs, particularly the loading dose, pose a challenge for immediate cost savings. The drug's dominance in terms of cost-effectiveness is evident only when considering a lifetime treatment horizon[3].

Competitive Landscape

ULTOMIRIS competes directly with eculizumab, another C5 complement inhibitor. However, its longer dosing interval and potential long-term cost savings position it favorably in the market. The drug's approval and positive clinical data have strengthened Alexion's market position in the rare disease segment[1][5].

Conclusion

ULTOMIRIS has marked a significant milestone in the treatment of PNH and other rare diseases, offering a more convenient and potentially cost-effective alternative to existing treatments. Alexion's strategic focus on driving conversion to ULTOMIRIS, expanding into new diseases, and diversifying its portfolio sets the stage for continued financial growth and market dominance.

Key Takeaways

• FDA Approval: ULTOMIRIS was approved by the FDA in December 2018 for adult patients with PNH.
• Clinical Efficacy: Demonstrated non-inferiority to eculizumab in phase 3 studies.
• Cost-Effectiveness: Potentially less costly over a lifetime treatment horizon but requires long-term treatment to realize savings.
• Financial Performance: Alexion has shown strong revenue and EPS growth, with a robust pipeline for future launches.
• Strategic Expansion: Focus on conversion to ULTOMIRIS, expansion into new diseases, and portfolio diversification.
• Safety and Tolerability: Well tolerated with a consistent safety profile across different indications.

FAQs

Q: What is ULTOMIRIS used for? A: ULTOMIRIS (ravulizumab-cwvz) is used for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and is also being studied for other rare diseases like generalized myasthenia gravis (gMG).

Q: How often is ULTOMIRIS administered? A: ULTOMIRIS is administered every eight weeks, following an initial loading dose.

Q: What are the key differences between ULTOMIRIS and eculizumab? A: ULTOMIRIS has a longer dosing interval (every eight weeks) compared to eculizumab (every two weeks), and it is potentially less costly over a lifetime treatment horizon.

Q: What are the most common adverse reactions associated with ULTOMIRIS? A: The most frequent adverse reactions include upper respiratory infection and headache.

Q: What is the financial outlook for Alexion Pharmaceuticals with ULTOMIRIS? A: Alexion has a strong financial outlook, with significant revenue and EPS growth, and a robust pipeline that includes multiple potential launches by 2023.

Sources

1. FDA approves ravulizumab-cwvz for paroxysmal nocturnal hemoglobinuria - FDA
2. Alexion Issues Statement on Path to Value Creation - Business Wire
3. Pharmacoeconomic Review - Ravulizumab (Ultomiris) - NCBI
4. Alexion Announces Positive Topline Results from Phase 3 Study of ULTOMIRIS® (ravulizumab-cwvz) in Adults with Generalized Myasthenia Gravis (gMG) - Alexion Pharmaceuticals
5. Alexion Receives Early FDA Approval for ULTOMIRIS (ravulizumab-cwvz) in Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) - BioSpace