Lonafarnib gets speedy review to treat progeria

June 10, 2020 0 By FM

EigerBio Pharmaceuticals Inc announced that the US FDA accepted the New Drug Application (NDA) for accelerated review of lonafarnib (Zokinvy) for the treatment of progeria and progeroid laminopathies.

The lonafarnib NDA includes data from a study which demonstrated a survival benefit with an 88% reduction in the risk of mortality in patients with progeria treated with lonafarnib monotherapy. 

The most commonly reported adverse events are gastrointestinal in nature. Many patients with progeria have received continuous lonafarnib therapy for greater than 10 years. There is currently no approved therapy for progeria or progeroid laminopathies.

Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is an ultra-rare and fatal genetic condition of accelerated aging in children. 

The condition is caused by a point mutation in the LMNA gene, which encodes the lamin A protein, yielding the farnesylated aberrant protein, progerin. 

Lonafarnib blocks the farnesylation of progerin and has been dosed in over 90 children with progeria at Boston Children’s Hospital in phase 1/2 and phase 2 studies. In patients with HGPS, lonafarnib monotherapy was associated with a lower mortality rate after 2.2 years of follow-up compared with no treatment (3.7% vs 33.3%, respectively) with a hazard ratio of 0.12 or a reduction in risk of mortality of 88%.