LONAFARNIB - Generic Drug Details

What are the generic sources for lonafarnib and what is the scope of patent protection?

Lonafarnib is the generic ingredient in one branded drug marketed by Sentynl Theraps Inc and is included in one NDA. There are two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Lonafarnib has nine patent family members in six countries.

Two suppliers are listed for this compound.

Summary for LONAFARNIB

International Patents:	9
US Patents:	2
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	2
Raw Ingredient (Bulk) Api Vendors:	73
Clinical Trials:	34
What excipients (inactive ingredients) are in LONAFARNIB?	LONAFARNIB excipients list
DailyMed Link:	LONAFARNIB at DailyMed

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

Constraining patent/regulatory exclusivity:

TREATMENT OF HUTCHINSON-GILFORD PROGERIA SYNDROME (HGPS) AND PROGEROID LAMINOPATHIES

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.

Recent Clinical Trials for LONAFARNIB

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

Sponsor	Phase
PRG Science & Technology Co., Ltd.	PHASE2
Soroka University Medical Center	Phase 3
Eiger BioPharmaceuticals	Phase 3

See all LONAFARNIB clinical trials

Pharmacology for LONAFARNIB

Drug Class	Farnesyltransferase Inhibitor
Mechanism of Action	Breast Cancer Resistance Protein Inhibitors Cytochrome P450 2C19 Inhibitors Cytochrome P450 2C8 Inhibitors Cytochrome P450 3A Inhibitors Farnesyltransferase Inhibitors Organic Anion Transporting Polypeptide 1B1 Inhibitors Organic Anion Transporting Polypeptide 1B3 Inhibitors P-Glycoprotein Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for LONAFARNIB

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for LONAFARNIB

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Sentynl Theraps Inc	ZOKINVY	lonafarnib	CAPSULE;ORAL	213969-001	Nov 20, 2020		RX	Yes	No
Sentynl Theraps Inc	ZOKINVY	lonafarnib	CAPSULE;ORAL	213969-001	Nov 20, 2020		RX	Yes	No
Sentynl Theraps Inc	ZOKINVY	lonafarnib	CAPSULE;ORAL	213969-002	Nov 20, 2020		RX	Yes	Yes	8,828,356

Expired US Patents for LONAFARNIB

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Sentynl Theraps Inc	ZOKINVY	lonafarnib	CAPSULE;ORAL	213969-002	Nov 20, 2020	8,828,356
Sentynl Theraps Inc	ZOKINVY	lonafarnib	CAPSULE;ORAL	213969-001	Nov 20, 2020	8,828,356

EU/EMA Drug Approvals for LONAFARNIB

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
EigerBio Europe Limited	Zokinvy	lonafarnib	EMEA/H/C/005271Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.	Authorised	no	no	yes	2022-07-18

International Patents for LONAFARNIB

Country	Patent Number	Title	Estimated Expiration
European Patent Office	1853265	INHIBITEURS DE FARNESYL TRANSFERASE DESTINES A TRAITER LES LAMINOPATHIES, LE VIEILLISSEMENT CELLULAIRE ET L'ATHEROSCLEROSE (FARNESYLTRANSFERASE INHIBITORS FOR USE IN THE TREATMENT OF LAMINOPATHIES, CELLULAR AGING AND ATHEROSCLEROSIS)
Japan	2006507845
World Intellectual Property Organization (WIPO)	2006081444

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: Lonafarnib

Last updated: July 28, 2025

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Introduction

Lonafarnib, a farnesyltransferase inhibitor (FTI), has garnered substantial attention within the pharmaceutical landscape owing to its unique mechanism targeting protein prenylation, particularly in the treatment of rare genetic disorders and certain cancers. Originally developed by Eiger BioPharmaceuticals and now under various clinical evaluations, lonafarnib’s market dynamics are shaped by its therapeutic potential, regulatory pathways, competitive landscape, and limited commercially approved indications. This analysis provides a comprehensive overview of the current and projected financial trajectory for lonafarnib, emphasizing its market drivers, challenges, and strategic outlook.

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Pharmacological Profile and Therapeutic Applications

Lonafarnib functions by inhibiting farnesyltransferase, an enzyme critical for post-translational modification of proteins such as Ras, which are implicated in cell proliferation and oncogenesis [1]. Its primary approved clinical application is in Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic disorder characterized by premature aging. The FDA approved lonafarnib in 2020 specifically for HGPS, marking a significant milestone given the lack of targeted therapies for this ultra-rare disease.

Additionally, lonafarnib is investigational for several oncological and infectious diseases, including hepatitis delta virus (HDV) infection, hepatocellular carcinoma, and certain leukemias. The compound’s repositioning potential, combined with its first-in-class status, consolidates its position in niche markets with high unmet needs.

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Market Drivers

1. Orphan Drug Designation and Regulatory Incentives
Lonafarnib’s designation as an orphan drug by agencies such as the FDA and EMA provides substantial advantages, including market exclusivity (7 years in the US), tax credits, and waived fees. These incentives significantly diminish R&D costs, encouraging continued investment and facilitating premium pricing strategies in ultra-rare indications like HGPS.

2. Rising Awareness and Diagnostics for Rare Diseases
Improved diagnostic capabilities and heightened awareness have increased identification rates for conditions like HGPS and HDV. This bolsters demand for lonafarnib and encourages healthcare providers to adopt targeted therapies.

3. Clinical Advancement in Hepatitis D Virus (HDV) Treatment
Lonafarnib’s promising efficacy in HDV offers sizeable commercial potential. Given the limited treatment options and high disease burden, successful clinical trial results are poised to open a new revenue stream. The recent positive phase 2 trials demonstrate improved antiviral responses, and ongoing phase 3 trials could pave the way for broader approval [2].

4. Strategic Partnerships and Collaborations
Eiger’s collaborations with biotech firms and academic institutions aim to expand lonafarnib’s therapeutic applications, accelerating development timelines and reducing costs. This strategic approach supports the drug’s financial growth prospects.

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Market Challenges and Risks

1. Limited Approved Indications and Small Patient Populations
The primary approved use for lonafarnib pertains to HGPS, which affects approximately 1 in 4 million to 8 million live births [3]. The ultra-rare nature constrains volume-based revenue growth, necessitating high per-patient pricing to sustain profitability.

2. Pricing and Reimbursement Hurdles
While orphan drugs command premium prices, payers are increasingly scrutinizing high-cost therapies, especially in the US. Negotiations and reimbursement policies could cap revenue potential.

3. Competitive Landscape
Emerging therapies targeting similar pathways or alternative treatment strategies for HGPS and HDV pose competitive risks. For instance, gene-editing approaches and antisense oligonucleotides are under exploration for genetic diseases, potentially challenging lonafarnib’s market share [4].

4. Regulatory and Clinical Trial Uncertainties
The success of ongoing trials, especially for HDV, hinges on regulatory acceptance of surrogate endpoints. Any setback could delay commercialization and impact financial projections.

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Financial Trajectory and Revenue Forecast

1. Revenues from Hutchinson-Gilford Progeria Syndrome
Eiger reports modest revenues attributable to lonafarnib, driven by niche patient access programs, compassionate use pathways, and limited commercial sales. The 2021 revenue was approximately $3.6 million, reflecting constrained market size but premium pricing [5].

2. Expansion through HDV and Other Indications
Positive phase 2 data in HDV position lonafarnib as a promising candidate for increased revenue in the upcoming years. Assuming phase 3 success and regulatory approval, revenues could escalate significantly, potentially reaching hundreds of millions annually. Industry estimates suggest a peak sales potential of $200-300 million for HDV alone, considering the global prevalence of hepatitis D (approximately 14 million chronic carriers) [6].

3. Pricing Strategy and Market Penetration
Premium pricing models, typical for orphan drugs, range from $150,000 to $250,000 annually per patient in the US. High-cost, low-volume sales necessitate strategic payor negotiations, partial government funding (e.g., via rare disease programs), and patient assistance schemes.

4. Long-Term Outlook and Revenue Growth
Market penetration prospects depend on trial outcomes and regulatory timelines. With ongoing clinical advancements, a conservative estimate anticipates a compound annual growth rate (CAGR) of 15-20% in revenues over the next 3-5 years, predominantly fueled by HDV and potentially expanding to other indications.

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Market Dynamics and Competitive Landscape

- Rare Disease Market:
HGPS treatment remains a highly specialized segment with limited competitors. The approval of lonafarnib creates a monopoly in this niche, though broader systemic therapies are in development.

- Viral Hepatitis Market:
The HDV segment presents significant growth opportunities with lonafarnib’s advancement. While interferon-based treatments have limited efficacy, lonafarnib’s targeted mechanism offers a promising alternative. Competition includes nucleotide/nucleoside analogs and novel agents such as Myrcludex B (bulevirtide), which received conditional marketing authorization in Europe.

- Oncology and Other Areas:
Despite ongoing research, lonafarnib remains investigational for cancer therapy. If clinical successes materialize, it could diversify revenue streams but will face a crowded anti-cancer pipeline.

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Strategic Outlook and Investment Considerations

Investors and stakeholders should monitor clinical trial results closely, particularly for HDV and other viral indications. The drug’s niche positioning for ultra-rare diseases affords high margins but limited volume. Strategic collaborations, regulatory milestones, and reimbursement policies significantly influence its financial path.

Long-term valuation hinges on successfully broadening indications and gaining access to larger patient populations through regulatory approvals. The current emphasis on rare and orphan drugs aligns favorably with lonafarnib’s profile, but the competitive landscape necessitates ongoing innovation and clinical validation.

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Key Takeaways

• Lonafarnib’s market strength stems from its orphan drug designation, targeting ultra-rare indications like HGPS, with high per-patient pricing mitigating volume constraints.
• The drug’s future growth potential is robust in the hepatitis delta virus (HDV) space, pending successful phase 3 trials and regulatory approval.
• Market challenges include limited patient populations, reimbursement hurdles, and emerging competitors in gene therapy and other treatment modalities.
• Financial projections anticipate a moderate revenue base for HGPS with significant upside from HDV expansion, potentially reaching hundreds of millions annually.
• Strategic partnerships, clinical trial success, and regulatory milestones are pivotal drivers of lonafarnib’s financial trajectory.

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FAQs

1. What is the current FDA-approved indication for lonafarnib?
Lonafarnib is FDA-approved for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic disorder causing premature aging.

2. How does lonafarnib’s orphan drug status impact its market prospects?
Orphan drug designation grants market exclusivity, tax incentives, and reduced development costs, enabling premium pricing and facilitating market entry in small patient populations.

3. What is the potential market size for lonafarnib in hepatitis delta virus infection?
Approximately 14 million individuals globally have chronic hepatitis D, offering a significant market opportunity if lonafarnib is approved for this indication.

4. What are the main risks associated with lonafarnib’s commercial success?
Risks include clinical trial failures, regulatory delays, reimbursement barriers, and competition from novel therapies such as gene editing and antiviral agents.

5. What strategic steps could enhance lonafarnib’s financial outlook?
Expansion into larger indications like HDV, strategic partnerships, obtaining regulatory approvals, and optimizing pricing and reimbursement strategies are critical.

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References

1. Khoury, K. et al. "Mechanism of action of lonafarnib in treating progeria." J Clin Invest. 2020.
2. Eiger BioPharmaceuticals, "Lonafarnib in the treatment of hepatitis delta virus: Phase 2 clinical trial." 2022.
3. Hennekam, R. C. M. "Hutchinson-Gilford progeria syndrome." Orphanet J Rare Dis. 2006.
4. Watanabe, M. et al. "Emerging gene therapies for rare genetic disorders." Nature Rev Drug Discov. 2021.
5. Eiger BioPharmaceuticals, Annual Report 2021.
6. World Health Organization. "Hepatitis D: Global prevalence and epidemiology." 2020.

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This comprehensive analysis underscores lonafarnib’s niche market strength and future growth avenues, emphasizing strategic actions that can maximize its financial trajectory within the pharmaceutical landscape.