The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for lonafarnib (Zokinvy™; Eiger BioPharmaceuticals) for the treatment of progeria and progeroid laminopathies, rare, genetic conditions of accelerated aging in children.
Lonafarnib is an oral inhibitor of farnesyltransferase, an enzyme involved in modification of proteins through a process called prenylation. It is believed to block the farnesylation of progerin protein.
The NDA includes data from a cohort study of 27 progeria patients treated with lonafarnib for a median duration of 2.2 years; outcomes from these patients were compared with those of 103 untreated patients from a separate study. Results showed that lonafarnib was associated with an 88% reduction in the risk of mortality when compared with no treatment (mortality rate: 3.7% vs 33.3%; HR, 0.12; 95% CI, 0.01-0.93; P =.04).
According to the Company, lonafarnib has been dosed in over 90 children with progeria at Boston Children’s Hospital in phase 1/2 and phase 2 studies.
A Prescription Drug User Fee Act (PDUFA) target date of November 20, 2020 has been set for the application. The FDA previously granted Orphan Drug and Breakthrough Therapy designations to lonafarnib for this indication.
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