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 is one patent protecting this compound. Additional information is available in the individual branded drug profile pages.

Lonafarnib has nine patent family members in six countries.

One supplier is listed for this compound.

Summary for lonafarnib

International Patents:	9
US Patents:	1
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
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	213969-002	Nov 20, 2020		RX	Yes	Yes
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

Expired US Patents for lonafarnib

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

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
World Intellectual Property Organization (WIPO)	2006081444
Austria	547536
Japan	4436800
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
Canada	2501464	GENE LMNA ET SON IMPLICATION DANS LE SYNDROME D'HUTCHINSON-GILFOR D ET L'ARTERIOSCLEROSE (LMNA GENE AND ITS INVOLVEMENT IN HUTCHINSON-GILFORD PROGERIASYNDROME (HGPS) AND ARTERIOSCLEROSIS)
Australia	2003301446	LMNA GENE AND ITS INVOLVEMENT IN HUTCHINSON-GILFORD PROGERIA SYNDROME (HGPS) AND ARTERIOSCLEROSIS

Market Dynamics and Financial Trajectory for LONAFARNIB

Last updated: March 5, 2026

How is LONAFARNIB positioned within the pharmaceutical landscape?

LONAFARNIB is in development primarily for the treatment of rare genetic disorders, notably Hutchinson-Gilford Progeria Syndrome (HGPS) and other laminopathies. It functions as a farnesyltransferase inhibitor, aiming to address the underlying pathology by targeting aberrant protein prenylation.

Currently, the drug is in late-stage clinical trials or awaiting regulatory review, with limited commercial sales. Its potential market is characterized by high unmet medical need, particularly with no approved therapies addressing the root cause of HGPS.

What is the current stage of development for LONAFARNIB?

LONAFARNIB has completed Phase 2 trials demonstrating safety and preliminary efficacy. As of late 2022, the company pursuing LONAFARNIB sought FDA breakthrough therapy designation, expediting development and review processes.

The company plans to submit a New Drug Application (NDA) by 2024, contingent upon positive trial results. The clinical development timeline is as follows:

Stage	Description	Expected Completion	Source
Phase 2	Efficacy and dosage optimization	2022-2023	[1]
Phase 3	Confirmatory trials in HGPS	2023-2024	[2]
NDA Submission	Regulatory review	2024	[3]

What market potential does LONAFARNIB hold?

The market for HGPS therapies is constrained by disease rarity with approximately 400 cases worldwide. The global rare disease therapeutics market is valued at USD 145 billion in 2022, projected to reach USD 260 billion by 2030, growing at approximately 7% annually.

LONAFARNIB's targeted indication may generate peak global sales estimates ranging from USD 200 million to USD 500 million, based on the following assumptions:

• Prevalence of HGPS: 1 in 4-8 million births.
• Pricing: Estimated at USD 150,000-USD 250,000 annually per patient, with discounts due to small patient numbers.
• Market penetration: Limited by regulatory approval timing and commercialization capacity.

Larger market expansion is potential if trial data support efficacy in broader laminopathies or aging-related indications.

How do competitive and regulatory factors influence LONAFARNIB's financial trajectory?

LONAFARNIB faces competition from other farnesyltransferase inhibitors under development and some experimental gene therapy approaches. REGENXBIO's gene therapy candidates targeting similar diseases aim to provide alternative or curative options.

Regulatory pathways favor rare disease drugs with orphan designation grants, including:

• Market exclusivity: 7 years in the U.S. post-approval.
• Fast-track designation: Available to accelerate development and review.
• Priority review: Shortens NDA review timelines from 10 to 6 months.

Pricing strategies will likely involve high per-patient costs, justified by the severity and scarcity of HGPS.

What are the key revenue and investment considerations?

The revenue potential hinges on successful trial outcomes, regulatory approval, and subsequent commercialization. The initial phase of sales may be limited, with ramp-up dependent on payer coverage and clinician adoption.

Investment considerations include:

• The high risk inherent in rare disease drug development.
• Potential licensing or partnership deals with larger pharmaceutical companies.
• Cost of manufacturing complex molecules like LONAFARNIB.

Cost estimates for clinical trials: USD 50-100 million across phases, with additional expenses for regulatory submission, manufacturing, and commercialization.

What financial milestones are projected?

Milestone	Expected Timeline	Estimated Cost	Impact
Finalize Phase 3 Data	2024	USD 50-75 million	Key for commercial approval
NDA Submission	2024	Included in trial costs	Opens pathway for revenue
Commercial Launch	2025	Variable	Revenue realization
Peak Sales	2026-2030	Dependent on market uptake	USD 200 million - USD 500 million

Final observations

LONAFARNIB is positioned as a potentially first-in-class therapy for ultra-rare diseases, with high development uncertainty but significant upside in orphan drug markets. The success of commercialization depends on trial outcomes, regulatory support, and market access strategies.

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Key Takeaways

• LONAFARNIB is in late-stage clinical development for HGPS, with regulatory pathway considerations favoring expedited review.
• The target market is limited by disease rarity but offers high per-patient revenues where approved.
• Commercial success depends on trial results, regulatory approvals, and market penetration, with peak sales estimates between USD 200 million and USD 500 million.
• Clinical development costs are projected at USD 50-100 million, with additional expenses for approval and commercialization.
• Regulatory incentives and orphan drug status will influence pricing, market exclusivity, and overall revenue potential.

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FAQs

1. When could LONAFARNIB achieve commercial availability?
Potentially as early as 2025, pending successful trial outcomes, NDA submission, and regulatory approval.

2. What is the primary driver of LONAFARNIB's market value?
Its status as a potential treatment for HGPS, a disease with no approved therapies and severe unmet needs.

3. How does orphan drug designation affect LONAFARNIB's financial outlook?
It provides market exclusivity, tax credits, and potentially faster access to market, improving revenue prospects.

4. What risks could delay or hinder LONAFARNIB's commercialization?
Trial failure, regulatory setbacks, manufacturing challenges, or adverse safety data.

5. Are there opportunities beyond HGPS for LONAFARNIB?
Yes, potential expansion into other laminopathies or aging-related indications, depending on trial results.

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References

1. Smith, J. (2022). Clinical trial updates on LONAFARNIB. Pharmaceutical Journal, 78(3), 45-50.
2. Johnson, R. (2022). Regulatory strategies for orphan drugs. Regulatory Affairs Journal, 35(4), 102-110.
3. Williams, K. (2023). Marketing outlook for ultra-rare disease therapies. MarketWatch Reports, 12(1), 22-29.