EIT Pharma, Inc announced that the US Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for lonafarnib for the treatment of chronic hepatitis D.

Chronic hepatitis D (CHD) is a serious, life-threatening liver disease caused by Hepatitis D Virus (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 US.

Dr. Leen Kawas, PhD, Chief Executive Officer (CEO), EIT Pharma, said, "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. We're grateful to the patients, investigators, and researchers whose commitment to the D-LIVR study made this milestone possible."

Lonafarnib is an investigational first-in-class oral CHD therapy candidate with a unique mechanism of action. Lonafarnib for CHD has previously received FDA Breakthrough Therapy Designation (BTD), Fast Track Designation (FTD), and Orphan Drug Designation (ODD).

Following its acquisition of the late-stage program in 2024, EIT Pharma worked closely with regulators and scientific and medical collaborators to complete the NDA submission and is now working on preparations for commercialisation. 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.

The NDA is supported by data from the phase 3 D-LIVR study, the largest clinical trial conducted to date in CHD, and phase 1/2 clinical data, nonclinical data, and manufacturing information. The global, randomised, placebo-controlled phase 3 D-LIVR study enrolled more than 400 patients across 21 countries and evaluated lonafarnib-based regimens over 48 weeks.

Dr. Jeffrey Glenn, Co-Founder, EIT Pharma and Joseph D. Grant, Professor and Professor of Medicine and Microbiology and Immunology, Stanford University, said, "Patients living with Chronic Hepatitis D urgently need novel therapies that target distinct steps in the HDV lifecycle. The FDA's acceptance of the NDA is an important first step toward 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 will continue to work collaboratively with the FDA during its review and will continue to prepare for the commercialisation of lonafarnib post-approval.