FDA Accepts EIT Pharma's NDA for Lonafarnib, First Oral Therapy Candidate for Chronic Hepatitis D
核心洞察
FDA has accepted for review EIT Pharma (搜索)'s New Drug Application for lonafarnib, a first-in-class oral therapy candidate for chronic hepatitis D (搜索), a serious liver disease with no currently approved oral treatments in the U.S.
The NDA is supported by data from the Phase 3 D-LIVR study, the largest clinical trial ever conducted in chronic hepatitis D (搜索), which enrolled over 400 patients across 21 countries.
Lonafarnib has received FDA Breakthrough Therapy, Fast Track, and Orphan Drug designations, and if approved would be the only oral late-stage therapy for this life-threatening condition.
EIT Pharma (搜索), Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for lonafarnib for the treatment of chronic hepatitis D (搜索) (CHD), marking a major milestone toward what could become the first oral treatment option for one of the most severe forms of viral hepatitis.
Chronic hepatitis D (搜索) is a serious, life-threatening liver disease caused by hepatitis D virus (搜索) (HDV) in individuals who already have hepatitis B virus infection. CHD can progress rapidly to cirrhosis, liver failure, liver cancer, and death. Despite the seriousness of the disease, treatment options remain limited, with no current FDA-approved oral therapies for CHD in the United States.
"FDA acceptance of our NDA is an important milestone in our mission to bring a potential new oral treatment to people living with chronic hepatitis D (搜索)," said Dr. Leen Kawas, PhD, Chief Executive Officer of EIT Pharma (搜索). "We're grateful to the patients, investigators, and researchers whose commitment to the D-LIVR study made this milestone possible."
D-LIVR: The Largest CHD Trial to Date
The NDA is supported by data from the Phase 3 D-LIVR study, the largest clinical trial conducted to date in chronic hepatitis D (搜索), along with Phase 1/2 clinical data, nonclinical data, and manufacturing information. The global, randomized, placebo-controlled Phase 3 D-LIVR study enrolled over 400 patients across 21 countries and evaluated lonafarnib-based regimens over 48 weeks.
Lonafarnib is an investigational first-in-class oral CHD therapy candidate with a unique mechanism of action. As an oral farnesyltransferase (搜索) inhibitor, lonafarnib is thought to exert antiviral activity by inhibiting the prenylation-dependent assembly process required for the formation of new HDV virions. It is currently the only oral therapeutic candidate in late-stage clinical development for chronic hepatitis D (搜索).
Regulatory Designations and Commercial Preparations
Lonafarnib for CHD has previously received FDA Breakthrough Therapy designation, Fast Track designation, and Orphan Drug designation. Following its acquisition of the late-stage program in 2024, EIT Pharma (搜索) worked closely with regulators and scientific and medical collaborators to complete the NDA submission and is now working on preparations for commercialization.
If approved, lonafarnib's oral administration, room-temperature storage, and potential for long-term event-free survival may offer practical advantages for people impacted by CHD and healthcare providers who consider treatment burden, duration and accessibility, and long-term clinical benefit when evaluating treatment options.
New Analyses Presented at EASL Congress 2026
At the EASL Congress 2026 held in Barcelona, Spain (May 27–30, 2026), EIT Pharma (搜索) presented new analyses from the lonafarnib development program. These included an oral presentation by Dr. Saeed Hamid demonstrating that lonafarnib/ritonavir and peg-interferon alfa (搜索) drive histological improvement and inflammation resolution in chronic hepatitis D (搜索), as well as a poster presentation showing that lonafarnib/ritonavir with or without peginterferon achieves rapid, durable responses.
"People living with chronic hepatitis D (搜索) face a serious and progressive liver disease with limited treatment options," said Dr. Kawas. "These new analyses from D-LIVR deepen our understanding of lonafarnib-based regimens for CHD treatment."
"Patients living with chronic hepatitis D (搜索) urgently need novel therapies that target distinct steps in the HDV lifecycle," said Dr. Jeffrey Glenn, co-founder of EIT Pharma (搜索) and Joseph D. Grant Professor and Professor of Medicine and Microbiology & Immunology at Stanford University. "The FDA's acceptance of the NDA is an important first step towards getting patients with CHD a new treatment option for this serious, life-threatening disease, and we look forward to working closely with the FDA throughout the review process."
EIT Pharma (搜索), based in Kirkland, Washington, was established by experienced clinical and biotechnology leaders to identify and advance overlooked or underdeveloped assets in areas of high unmet medical need. Under Dr. Kawas's leadership, the company reinitiated development of lonafarnib for CHD after prior development activity had stopped, securing the rights to the program and the capital to restart work. Just over a year after restarting the program, EIT Pharma has defined a potential regulatory path forward and continues to prepare for commercialization.
The company expects to continue working collaboratively with the FDA during its review. Lonafarnib has not been approved by the FDA or any other regulatory authority for the treatment of chronic hepatitis D (搜索), and its safety and efficacy have not been established.