Eiger BioPharmaceuticals has begun a rolling New Drug Application (NDA) for lonafarnib, for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies. 

The Food and Drug Administration (FDA) agreed that this submission could be part of a Rolling Review Process, allowing completed portions of an NDA to be submitted and reviewed on an ongoing basis. 

Lonafarnib is an oral farnesyltransferase inhibitor; farnesyltransferase is an enzyme involved in modification of proteins through a process called prenylation. Lonafarnib is believed to block farnesylation of progerin. In a cohort study, 27 progeria patients were treated with lonafarnib monotherapy and compared to 103 untreated progeria patients, for a median of 2.2 years of follow up. Results found that treatment with lonafarnib was associated with a significantly lower mortality rate compared with no treatment (3.7% vs 33.3%; HR, 0.12; 95% CI, 0.01-0.93; P =.04). 

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Children with Progeria die of heart disease at an average age of 14.5 years. “Eiger is committed to making a meaningful difference in the lives of patients with Progeria and Progeroid Laminopathies and we are thrilled to submit our first NDA for these children,” said David Cory, Eiger President and CEO. “We look forward to working closely with the FDA as we complete the rolling NDA process.”

Lonafarnib has previously been granted Orphan Drug and Breakthrough Therapy Designation for Progeria by the FDA.

For more information visit eigerbio.com.