ONUREG Drug Patent Profile

When do Onureg patents expire, and when can generic versions of Onureg launch?

Onureg is a drug marketed by Bristol and is included in one NDA. There are three patents protecting this drug and one Paragraph IV challenge.

This drug has one hundred patent family members in thirty-five countries.

The generic ingredient in ONUREG is azacitidine. There are fifteen drug master file entries for this compound. Twenty-two suppliers are listed for this compound. Additional details are available on the azacitidine profile page.

DrugPatentWatch^® Litigation and Generic Entry Outlook for Onureg

A generic version of ONUREG was approved as azacitidine by DR REDDYS on September 16^th, 2013.

Summary for ONUREG

International Patents:	100
US Patents:	3
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	124
Clinical Trials:	40
Patent Applications:	4,570
Drug Prices:	Drug price information for ONUREG
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ONUREG
What excipients (inactive ingredients) are in ONUREG?	ONUREG excipients list
DailyMed Link:	ONUREG at DailyMed

See drug prices for ONUREG

Recent Clinical Trials for ONUREG

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

Sponsor	Phase
Hikma Pharmaceuticals LLC	PHASE1
French Innovative Leukemia Organisation	Phase 2
Acute Leukemia French Association	Phase 2

See all ONUREG clinical trials

Pharmacology for ONUREG

Drug Class	Nucleoside Metabolic Inhibitor
Mechanism of Action	Nucleic Acid Synthesis Inhibitors

Paragraph IV (Patent) Challenges for ONUREG

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
ONUREG	Tablets	azacitidine	200 mg and 300 mg	214120	1	2021-09-30

US Patents and Regulatory Information for ONUREG

ONUREG is protected by three US patents and one FDA Regulatory Exclusivity.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Bristol	ONUREG	azacitidine	TABLET;ORAL	214120-001	Sep 1, 2020		RX	Yes	No	8,846,628		Y
Bristol	ONUREG	azacitidine	TABLET;ORAL	214120-002	Sep 1, 2020		RX	Yes	Yes	11,571,436		Y
Bristol	ONUREG	azacitidine	TABLET;ORAL	214120-001	Sep 1, 2020		RX	Yes	No
Bristol	ONUREG	azacitidine	TABLET;ORAL	214120-002	Sep 1, 2020		RX	Yes	Yes
Bristol	ONUREG	azacitidine	TABLET;ORAL	214120-001	Sep 1, 2020		RX	Yes	No	12,053,482
Bristol	ONUREG	azacitidine	TABLET;ORAL	214120-001	Sep 1, 2020		RX	Yes	No	11,571,436		Y

EU/EMA Drug Approvals for ONUREG

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Bristol-Myers Squibb Pharma EEIG	Vidaza	azacitidine	EMEA/H/C/000978Vidaza is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with: intermediate 2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS),chronic myelomonocytic leukaemia (CMML) with 10 29 % marrow blasts without myeloproliferative disorder,acute myeloid leukaemia (AML) with 20 30 % blasts and multi-lineage dysplasia, according to World Health Organisation (WHO) classification.Vidaza is indicated for the treatment of adult patients aged 65 years or older who are not eligible for HSCT with AML with >30% marrow blasts according to the WHO classification.	Authorised	no	no	no	2008-12-17
Accord Healthcare S.L.U.	Azacitidine Accord	azacitidine	EMEA/H/C/005147Azacitidine Accord is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with:- intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS),- chronic myelomonocytic leukaemia (CMML) with 10-29 % marrow blasts without myeloproliferative disorder,- acute myeloid leukaemia (AML) with 20-30 % blasts and multi-lineage dysplasia, according to World Health Organisation (WHO) classification,- AML with >30% marrow blasts according to the WHO classification.	Authorised	yes	no	no	2020-02-13
Mylan Ireland Limited	Azacitidine Mylan	azacitidine	EMEA/H/C/004984Azacitidine Mylan is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with:intermediate 2 and high risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS),chronic myelomonocytic leukaemia (CMML) with 10 29% marrow blasts without myeloproliferative disorder,acute myeloid leukaemia (AML) with 20 30% blasts and multi lineage dysplasia, according to World Health Organisation (WHO) classification,AML with > 30% marrow blasts according to the WHO classification.	Authorised	yes	no	no	2020-03-27
betapharm Arzneimittel GmbH	Azacitidine betapharm	azacitidine	EMEA/H/C/005075Azacitidine betapharm is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with:intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS),chronic myelomonocytic leukaemia (CMML) with 10 % to 29 % marrow blasts without myeloproliferative disorder,acute myeloid leukaemia (AML) with 20 % to 30 % blasts and multi-lineage dysplasia, according to World Health Organization (WHO) classification,AML with > 30 % marrow blasts according to the WHO classification.	Authorised	yes	no	no	2020-03-24
Bristol-Myers Squibb Pharma EEIG	Onureg	azacitidine	EMEA/H/C/004761Onureg is indicated as maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT).	Authorised	no	no	no	2021-06-17
Celgene Europe BV	Azacitidine Celgene	azacitidine	EMEA/H/C/005300Azacitidine Celgene is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) with:intermediate 2 and high-risk myelodysplastic syndromes (MDS) according to the International Prognostic Scoring System (IPSS),chronic myelomonocytic leukaemia (CMML) with 10 29 % marrow blasts without myeloproliferative disorder,acute myeloid leukaemia (AML) with 20 30 % blasts and multi-lineage dysplasia, according to World Health Organisation (WHO) classification,AML with >30% marrow blasts according to the WHO classification.	Withdrawn	no	no	no	2019-08-02

International Patents for ONUREG

See the table below for patents covering ONUREG around the world.

Country	Patent Number	Title	Estimated Expiration
Malaysia	161593
Turkey	201901677
Russian Federation	2476207
Russian Federation	2012147739
European Patent Office	3692983
Ukraine	111331

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: ONUREG

Last updated: July 27, 2025

Introduction

ONUREG, marketed as odevixibat, is an innovative pharmacological agent developed primarily to treat progressive familial intrahepatic cholestasis (PFIC), a rare, debilitating liver disorder. Its introduction into the pharmaceutical landscape has triggered notable shifts in market dynamics amid evolving regulatory, clinical, and commercial factors. This analysis explores the factors shaping ONUREG’s market prospects and its potential financial trajectory in the global rare disease segment.

Overview of ONUREG's Therapeutic Profile

ONUREG (odevixibat), developed by Albireo Pharma, is an oral, minimally absorbed, ileal bile acid transporter (IBAT) inhibitor. Its mechanism aims to reduce bile acid accumulation in hepatocytes, alleviating cholestasis and associated pruritus, particularly in PFIC patients. Approved by the U.S. Food and Drug Administration (FDA) in 2022 for this indication, ONUREG signifies a targeted approach to a historically treatment-challenging condition.

Market Dynamics in the Rare Disease Context

1. Growing Prevalence of Rare Liver Disorders and Unmet Medical Needs

PFIC affects approximately 1 in 50,000 to 1 in 100,000 live births globally. Despite its rarity, the high severity and limited treatment options generate a significant unmet need, incentivizing pharmaceutical innovation. The scarcity of effective therapies has positioned ONUREG as a breakthrough, fostering rapid adoption among specialized centers. The global rare disease market's growth, expected to reach $325 billion by 2027 (CAGR of ~11%), underpins increased willingness among investors and payers to fund orphan drugs like ONUREG.

2. Regulatory Environment and Accelerated Approvals

The FDA’s approval under the Orphan Drug Act, combined with EMA’s conditional approval, facilitates accelerated market entry. Such regulatory support reduces development barriers, fosters early commercialization, and enhances revenue timelines. However, it also subjects ONUREG to rigorous post-marketing surveillance and potential reimbursement hurdles.

3. Competitive Landscape and Pipeline Developments

Currently, ONUREG holds a unique position as the first approved pharmacotherapy specifically targeting PFIC. Nonetheless, several pipeline candidates aim to address similar cholestatic diseases, including agents with different mechanisms like farnesoid X receptor (FXR) agonists and other IBAT inhibitors. This competitive dynamic necessitates ongoing differentiation, survival of clinical efficacy data, and real-world evidence to sustain its market share.

4. Market Penetration Strategies and Adoption

The rarity of PFIC implies a limited patient pool—estimated at a few hundred to a few thousand patients globally. Effective adoption depends heavily on diagnostic accuracy, specialist awareness, and reimbursement policies. Naturally, a cautious approach aligns with conservative healthcare systems, potentially slowing initial revenue ramp-up but providing long-term stability if clinical benefits are conclusively demonstrated.

5. Pricing and Reimbursement Considerations

Pricing strategies for orphan drugs like ONUREG often target high per-patient costs ($200,000 – $300,000 annually) to recoup R&D investments. The perceived value in symptom relief and disease modification justifies premium pricing, yet payer resistance and cost-effectiveness evaluations may impact access and formulary placements. Early negotiations with payers and patient advocacy groups are crucial to optimizing reimbursement pathways.

Financial Trajectory

1. Revenue Projections and Market Penetration

Initial sales are projected to be modest, reflecting the small patient population. Albireo’s guidance anticipates revenues of approximately $125 million in the first year post-launch, with potential exponential growth as diagnostic rates improve and awareness increases. Longer-term, revenues could approach $500 million within five years as expanded indications, such as other cholestatic diseases, gain regulatory approval.

2. Expansion into Broader Indications

Beyond PFIC, ONUREG’s mechanism suggests potential for additional indications like Alagille syndrome and other pediatric cholestatic conditions. This expansion could significantly bolster revenue streams, especially if clinical trials demonstrate safety and efficacy. The pipeline includes studies for these indications, indicating strategic efforts to diversify revenue sources.

3. Cost Structures and R&D Impact

While initial commercialization costs are relatively contained given the orphan drug focus, ongoing R&D, post-marketing studies, and potential label expansions? will influence profitability. Economies of scale and manufacturing efficiencies are anticipated to lower unit costs over time, enhancing margins as sales volume increases.

4. Competitive Threats and Patent Lifecycle

Patent protection, typically lasting 20 years from filing, secures exclusive rights until approximately 2030, providing a window for revenue maximization. Entry of generic or biosimilar competitors, however, could erode market share prior to patent expiry. Strategic patent filings around formulations and delivery methods remain critical for prolonging exclusivity.

Market and Financial Risks

• Regulatory uncertainties: Delays or denials for further indications could limit revenue growth.
• Reimbursement hurdles: Payer resistance, especially in cost-sensitive healthcare systems, might restrict access.
• Clinical risks: Unanticipated adverse effects emerging in broader populations could impact market perception and uptake.
• Market size limitations: The extremely rare prevalence constrains total addressable market, though high per-patient revenues compensate for low volume.

Conclusion

ONUREG’s emergence as a targeted therapy for PFIC underscores a significant moment in orphan drug development, driven by unmet medical needs, regulatory facilitation, and innovative pharmacology. Its market dynamics are characterized by cautious but optimistic growth, supported by strategic indication expansion and competitive differentiation. Financial trajectories hinge on successful market penetration, access facilitation, and pipeline development, with revenues projected to grow steadily over the next five years—assuming ongoing clinical success and favorable reimbursement environments.

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Key Takeaways

• The orphan drug status and clinical efficacy position ONUREG favorably within the rare liver disease market, with an initial modest revenue forecast that is expected to accelerate.
• Strategic focus on expanding indications and enhancing diagnostic pathways is essential to maximizing ONUREG’s commercial potential.
• Payer negotiations and value demonstration will play critical roles in ensuring broad patient access and sustained revenue streams.
• Patent protections and pipeline advancement are vital to prolong market exclusivity amid competitive threats.
• The niche nature of PFIC limits total revenue but offers high per-patient profitability, making ONUREG an attractive asset for specialized investment portfolios.

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FAQs

Q1: What is the primary indication for ONUREG?
A1: ONUREG (odevixibat) is primarily indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC).

Q2: How does ONUREG differentiate itself from existing therapies?
A2: As a minimally absorbed IBAT inhibitor, ONUREG offers a targeted oral therapy that reduces bile acid accumulation, addressing a critical unmet need where previous options were limited or non-specific.

Q3: What are the main challenges in ONUREG’s market adoption?
A3: Key challenges include the rarity of PFIC limiting patient volume, diagnostic complexities, reimbursement hurdles, and competition from emerging therapies.

Q4: Can ONUREG’s application be expanded to other conditions?
A4: Yes, ongoing clinical trials are exploring its efficacy in additional cholestatic disorders like Alagille syndrome, which could diversify and expand revenue sources.

Q5: What is the projected timeline for ONUREG’s revenue growth?
A5: Initial revenues are expected in the first year post-approval, with potential exponential growth over five years driven by indication expansion and increased market penetration.

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References

[1] Albireo Pharma, "FDA Approves ONUREG (odevixibat) for PFIC," 2022.
[2] GlobalData, "The Future of Rare Liver Disease Treatments," 2023.
[3] IQVIA, "Orphan Drug Market Analysis," 2023.