DEFIBROTIDE SODIUM - Generic Drug Details

What are the generic drug sources for defibrotide sodium and what is the scope of freedom to operate?

Defibrotide sodium is the generic ingredient in one branded drug marketed by Jazz Pharms Inc and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Defibrotide sodium has twenty-five patent family members in sixteen countries.

One supplier is listed for this compound.

Summary for DEFIBROTIDE SODIUM

International Patents:	25
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	9
Clinical Trials:	1
What excipients (inactive ingredients) are in DEFIBROTIDE SODIUM?	DEFIBROTIDE SODIUM excipients list
DailyMed Link:	DEFIBROTIDE SODIUM at DailyMed

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

Constraining patent/regulatory exclusivity:

11,236,328

Dosage:

SOLUTION;INTRAVENOUS

^*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.

Recent Clinical Trials for DEFIBROTIDE SODIUM

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
University of California, San Francisco	Phase 2
Jazz Pharmaceuticals	Phase 2

See all DEFIBROTIDE SODIUM clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for DEFIBROTIDE SODIUM

B01AX	Other antithrombotic agents
B01A	ANTITHROMBOTIC AGENTS
B01	ANTITHROMBOTIC AGENTS
B	Blood and blood forming organs

US Patents and Regulatory Information for DEFIBROTIDE SODIUM

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes	11,236,328
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes	11,746,348		Y
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes	11,085,043		Y

International Patents for DEFIBROTIDE SODIUM

Country	Patent Number	Title	Estimated Expiration
Japan	2015521477	デフィブロチドの生物学的活性を決定する為の、ユーグロブリンに基づく方法
European Patent Office	2864496	PROCÉDÉ À BASE D'EUGLOBULINE POUR LA DÉTERMINATION DE L'ACTIVITÉ BIOLOGIQUE DE DÉFIBROTIDE (EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE)
South Korea	20180098420	디파이브로타이드의 생물학적 활성 측정을 위한 유우글로불린에 기초한 방법 (EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE)
Hong Kong	1208503	用於測定去纖維蛋白多核苷酸的生物活性的基於優球蛋白的方法 (EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE)
Russian Federation	2014149089	СПОСОБ ОПРЕДЕЛЕНИЯ БИОЛОГИЧЕСКОЙ АКТИВНОСТИ ДЕФИБРОТИДА, ОСНОВАННЫЙ НА ПРИМЕНЕНИИ ЭУГЛОБУЛИНА
Australia	2012383169	Euglobulin-based method for determining the biological activity of defibrotide

DEFIBROTIDE SODIUM Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Defibrotide Sodium

Introduction

Defibrotide sodium, marketed as Defitelio, is a critical medication approved for the treatment of hepatic veno-occlusive disease (VOD), also known as sinusoidal obstructive syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT). Here, we delve into the market dynamics and financial trajectory of this life-saving drug.

Approval and Indications

Defibrotide sodium was approved by the U.S. Food and Drug Administration (FDA) on March 30, 2016, for the treatment of adult and pediatric patients with hepatic VOD and associated multi-organ dysfunction[1][4].

Clinical Efficacy

The efficacy of defibrotide sodium was established through three studies, including two prospective clinical trials and an expanded access study, involving 528 patients. These studies demonstrated significant improvements in Day +100 survival rates compared to supportive care or other interventions, with survival rates ranging from 38% to 45% in the treatment groups versus 21% to 31% in the control groups[1].

Market Growth

The defibrotide market has experienced rapid and substantial growth in recent years. Projections indicate continued significant expansion from 2023 to 2031, driven by the increasing demand for effective treatments for VOD/SOS and the lack of alternative approved therapies[3].

Revenue Performance

Jazz Pharmaceuticals, the manufacturer of Defitelio, has reported steady revenue growth from the drug. In 2018, Defitelio net product sales increased by 12% compared to the previous year, and by 4% in the fourth quarter of 2018[2].

In more recent financial reports, Defitelio continued to contribute significantly to Jazz Pharmaceuticals' revenue. For 2023, Defitelio net product sales were $184 million, although this represented a slight decline from $194 million in 2022[5].

Global Availability

Defitelio is approved and available in several countries, including the U.S., Canada, Europe, Israel, and South Korea. The drug's availability in these markets has expanded its reach and contributed to its revenue growth[4].

Safety and Adverse Reactions

While defibrotide sodium is a life-saving treatment, it comes with potential risks. The drug is contraindicated in patients receiving anticoagulants or fibrinolytic therapies due to its profibrinolytic activity. Common adverse reactions include hypotension, diarrhea, vomiting, nausea, and epistaxis. Serious adverse events such as hemorrhage and hypersensitivity reactions have also been reported[1].

Market Dynamics

The market for defibrotide sodium is driven by several key factors:

Unmet Medical Need

VOD/SOS is a devastating condition with limited treatment options, making defibrotide sodium a critical therapeutic choice. The approval of Defitelio addressed a significant unmet medical need, contributing to its market success[4].

Regulatory Support

The drug received priority review status in several jurisdictions, including the U.S. and Canada, due to its potential to treat a serious and life-threatening condition. This expedited approval process facilitated its entry into the market[1][4].

Competitive Landscape

Defitelio currently holds a unique position in the market as the only approved treatment for VOD/SOS with renal or pulmonary dysfunction following HSCT. This lack of competition has been a significant factor in its revenue growth[3].

Financial Projections

The financial trajectory for defibrotide sodium is positive, with projections indicating continued growth. The global defibrotide market is expected to expand significantly from 2023 to 2031, driven by increasing demand and the absence of alternative treatments[3].

Jazz Pharmaceuticals' financial guidance for 2024 reflects continued top-line growth, although the specific contribution of Defitelio to this growth is expected to be offset by declines in other product lines[5].

Conclusion

Defibrotide sodium, marketed as Defitelio, has established itself as a crucial treatment for hepatic VOD/SOS, driving significant revenue growth for Jazz Pharmaceuticals. The drug's unique market position, regulatory support, and unmet medical need have contributed to its success. As the global market continues to expand, Defitelio is poised to remain a key player in the treatment of this life-threatening condition.

Key Takeaways

• Approval and Indications: Defibrotide sodium is approved for treating hepatic VOD/SOS with renal or pulmonary dysfunction following HSCT.
• Clinical Efficacy: Demonstrated significant improvements in Day +100 survival rates.
• Market Growth: Rapid and substantial growth projected from 2023 to 2031.
• Revenue Performance: Steady revenue growth, though with some recent decline.
• Global Availability: Approved in multiple countries, including the U.S., Canada, Europe, Israel, and South Korea.
• Safety and Adverse Reactions: Potential risks include hemorrhage and hypersensitivity reactions.
• Market Dynamics: Driven by unmet medical need, regulatory support, and lack of competition.
• Financial Projections: Continued growth expected, though with some offset by declines in other product lines.

FAQs

What is Defitelio used for?

Defitelio (defibrotide sodium) is used for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstructive syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)[1].

What are the common adverse reactions associated with Defitelio?

Common adverse reactions include hypotension, diarrhea, vomiting, nausea, and epistaxis. Serious adverse events such as hemorrhage and hypersensitivity reactions have also been reported[1].

In which countries is Defitelio approved?

Defitelio is approved in the U.S., Canada, Europe, Israel, and South Korea[4].

What is the recommended dose and schedule for Defitelio?

The recommended dose is 6.25 mg/kg intravenously every 6 hours, given as a 2-hour infusion for at least 21 days, and continued until VOD resolution or up to 60 days of treatment[1].

How has Defitelio contributed to Jazz Pharmaceuticals' revenue?

Defitelio has been a significant contributor to Jazz Pharmaceuticals' revenue, with steady growth reported in recent years, although there has been a slight decline in some periods[2][5].

What are the market projections for Defitelio?

The global defibrotide market is expected to expand significantly from 2023 to 2031, driven by increasing demand and the absence of alternative treatments[3].