Eiger BioPharmaceuticals announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome (HGPS or progeria) and progeroid laminopathies.
There is currently no approved treatment for progeria, an ultra-rare and rapidly fatal genetic condition that causes accelerated aging in children. Lonafarnib, a first-in-class prenylation inhibitor, works by inhibiting the farnesyltransferase enzyme that is involved in protein modification; it blocks the farnesylation of the progerin protein.
In Phase 1/2 and Phase 2 clinical studies, lonafarnib was associated with statistically significant improvements in survival. Pooled data of 63 patients showed a 77% reduction in mortality risk in patients treated with lonafarnib monotherapy vs matched untreated controls over an average follow-up of 2.2 years (hazard ratio [HR] 0.23; P =.04).
The Company anticipates a New Drug Application (NDA) submission to the FDA in 2019.
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