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.

One supplier is listed for this compound.

Summary for LONAFARNIB

International Patents:	9
US Patents:	2
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	73
Clinical Trials:	33
Patent Applications:	4,305
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
Soroka University Medical Center	Phase 3
Eiger BioPharmaceuticals	Phase 3
Boston Children’s Hospital	Phase 1/Phase 2

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
Sentynl Theraps Inc	ZOKINVY	lonafarnib	CAPSULE;ORAL	213969-001	Nov 20, 2020		RX	Yes	No

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/005271 Zokinvy 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)	2004035753
Japan	4436800
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)
Austria	547536
European Patent Office	1552020	GENE LMNA ET SON IMPLICATION DANS LE SYNDROME D'HUTCHINSON-GILFORD ET L'ARTERIOSCLEROSE (LMNA GENE AND ITS INVOLVEMENT IN HUTCHINSON-GILFORD PROGERIA SYNDROME (HGPS) AND ARTERIOSCLEROSIS)

LONAFARNIB Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Lonafarnib

Introduction

Lonafarnib, a first-in-class prenylation inhibitor, has been at the forefront of innovative therapies for several severe and rare diseases, including hepatitis delta virus (HDV) and Hutchinson-Gilford progeria syndrome (HGPS). Here, we delve into the market dynamics and financial trajectory of lonafarnib, highlighting its development, regulatory milestones, and financial implications.

Development and Clinical Trials

Hepatitis Delta Virus (HDV)

Lonafarnib is being developed as a treatment for HDV, a condition with no FDA-approved therapy. The Phase 3 D-LIVR study has been pivotal in this endeavor. This global, multi-center study evaluates two lonafarnib-based treatments: an all-oral regimen of lonafarnib boosted with ritonavir and a combination regimen of lonafarnib boosted with ritonavir combined with peginterferon alfa. Both regimens have shown statistically significant results in achieving the composite primary endpoint of a ≥2 log decline in HDV RNA and normalization of alanine aminotransferase (ALT) at Week 48 compared to placebo[4].

Clinical Outcomes

The D-LIVR study results are promising, with response rates of 10.1% for the all-oral regimen and 19.2% for the combination regimen, both statistically significant against placebo. These outcomes indicate a potential breakthrough in the treatment of HDV, a disease with high unmet medical needs[4].

Regulatory Milestones

Pre-NDA Meeting

Eiger BioPharmaceuticals plans to engage with regulatory agencies, starting with a pre-NDA meeting with the FDA anticipated by the end of Q2 2023, to discuss pathways for regulatory submissions. This meeting is a critical step towards potential marketing approval for lonafarnib-based treatments for HDV[1][4].

Orphan Drug Designation

Lonafarnib has been granted Orphan Drug designation by the U.S. FDA for the treatment of chronic HDV, which provides certain benefits such as tax credits, user fee waivers, and market exclusivity if approved[4].

Financial Trajectory

Research and Development Expenses

Eiger BioPharmaceuticals has incurred significant research and development expenses related to the development of lonafarnib. For the full year 2022, these expenses totaled $75.3 million, driven by increases in headcount, clinical and manufacturing expenditures, and milestones related to the Phase 3 LIMT-2 study of peginterferon lambda[1].

Revenue and Net Loss

Despite the progress in clinical trials, Eiger BioPharmaceuticals has historically incurred operating losses. For 2022, the company reported a net loss of $96.8 million. However, the commercial launch of Zokinvy (lonafarnib) for HGPS in the U.S. in January 2021 has started generating product revenue, with $12.7 million in product revenue for 2022[3].

Cash Position

As of December 31, 2022, Eiger BioPharmaceuticals had $98.9 million in cash, cash equivalents, and short-term debt securities. This financial position is crucial for continuing the clinical development and potential commercialization of lonafarnib-based treatments[1].

Market Dynamics

Acquisition and Licensing

In a significant market move, Sentynl Therapeutics, a Zydus Group company, acquired the global rights to Zokinvy (lonafarnib) from Eiger BioPharmaceuticals for the treatment of HGPS. This acquisition expands Sentynl's portfolio of biopharmaceuticals for rare and ultra-rare diseases and underscores the value of lonafarnib in addressing unmet medical needs[2].

Competitive Landscape

The market for HDV treatments is currently devoid of FDA-approved therapies, making lonafarnib a potential game-changer. Eiger's focus on developing innovative therapies positions the company to capture a significant market share if lonafarnib receives regulatory approval[3].

Patient and Market Impact

Lonafarnib's potential approval could have a profound impact on patients with HDV, offering a treatment option where none currently exists. The drug's efficacy in reducing HDV viral load and improving liver function could improve patient outcomes and quality of life, thereby driving market demand[4].

Future Outlook

Regulatory Approval and Commercialization

The success of the D-LIVR study and upcoming regulatory meetings set the stage for potential FDA approval. If approved, lonafarnib could become a cornerstone in the treatment of HDV, driving significant revenue growth for Eiger BioPharmaceuticals or its future owners of the drug's rights.

Continued Research and Development

Eiger BioPharmaceuticals is committed to advancing its product candidates, including lonafarnib. The company's ongoing research and development efforts are aimed at enhancing the efficacy and safety profile of lonafarnib-based treatments, which could lead to expanded indications and further market penetration.

Key Takeaways

• Clinical Success: Lonafarnib has shown statistically significant results in the Phase 3 D-LIVR study for HDV.
• Regulatory Milestones: A pre-NDA meeting with the FDA is planned, marking a crucial step towards potential marketing approval.
• Financial Position: Eiger BioPharmaceuticals has incurred significant R&D expenses but maintains a strong cash position to support ongoing development.
• Market Dynamics: The acquisition of Zokinvy by Sentynl Therapeutics highlights the drug's value, and potential FDA approval could capture a significant market share.
• Future Outlook: Regulatory approval and commercialization could drive substantial revenue growth and improve patient outcomes.

FAQs

Q: What is the current status of lonafarnib in clinical trials for HDV?

A: Lonafarnib is in the Phase 3 D-LIVR study, which has shown statistically significant results in achieving the composite primary endpoint. A pre-NDA meeting with the FDA is planned by the end of Q2 2023[1][4].

Q: What are the potential treatment regimens being evaluated for HDV?

A: The D-LIVR study evaluates two regimens: an all-oral regimen of lonafarnib boosted with ritonavir and a combination regimen of lonafarnib boosted with ritonavir combined with peginterferon alfa[4].

Q: Has lonafarnib received any regulatory designations?

A: Yes, lonafarnib has been granted Orphan Drug designation by the U.S. FDA for the treatment of chronic HDV[4].

Q: Who acquired the global rights to Zokinvy (lonafarnib) for HGPS?

A: Sentynl Therapeutics, a Zydus Group company, acquired the global rights to Zokinvy from Eiger BioPharmaceuticals[2].

Q: What is the financial outlook for Eiger BioPharmaceuticals related to lonafarnib?

A: Eiger BioPharmaceuticals has incurred significant R&D expenses but maintains a strong cash position. Potential FDA approval and commercialization of lonafarnib could drive substantial revenue growth[1][3].

Sources

1. Eiger BioPharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update - PR Newswire
2. Sentynl Therapeutics Announces Global Acquisition of Zokinvy (Lonafarnib) for Treatment of Hutchinson-Gilford Progeria Syndrome from Eiger BioPharmaceuticals - Sentynl Therapeutics
3. Eiger BioPharmaceuticals, Inc. 10K 2022 - Eiger BioPharmaceuticals
4. Eiger Announces Both Lonafarnib-based Treatments in Pivotal Phase 3 D-LIVR Trial in Hepatitis Delta Virus (HDV) Achieved Statistical Significance Against Placebo in Composite Primary Endpoint - PR Newswire
5. The farnesyl transferase inhibitor (FTI) lonafarnib improves nuclear morphology of HGPS patient cells - Taylor & Francis Online