According to a publication from Pharmaceutical Technology, California-based Eiger Biopharmaceuticals is preparing to submit a New Drug Application (NDA) to the Food and Drug Administration (FDA) for their experimental progeria drug lonafarnib. The New Drug Application is the final step of bringing a drug to market in the United States.
Lonafarnib has been designated as an orphan drug, breakthrough therapy, and rare pediatric disease medicine. Each designation brings with it certain perks that ease the burden of research and development — many of which are money-related.
About Progeria
Progeria, or Hutchinson-Gilford progeria more properly, is an extremely rare genetic condition characterized by the appearance of rapid and dramatic aging in early childhood. Affected individuals often develop a characteristic facial appearance — including prominent eyes, thin nose, and protruding ears. Individuals frequently fail to thrive (insufficient weight gain), and are likely to develop hardened arteries that can increase the mortality risk of vascular events.
The “aging” appearance of progeria is caused by a single genetic mutation to the LMNA gene. Normally, LMNA codes for the production of a protein called Lamin A/C — an important structural protein in cells. Lamin A/C is one of the proteins responsible for binding the nucleus together.
When LMNA mutates, it provides faulty instructions for Lamin A/C production. Instead, the body produces progerin — a mutant form of the protein that is nowhere near as effective at providing structure to the nucleus. These destabilized nuclei are the cause of progeria’s “rapid aging” symptoms, and the multitude of serious complications that can accompany it.
Individuals with progeria have reduced life expectancy — though they have lived into their 20s, most do not survive all of adolescence.
Lonafarnib Inching Towards New Drug Application
Lonafarnib was first brought to clinical trial over a decade ago in 2007. It was found to bring about significant weight increase in progeria patients who received the farnesyltransferase inhibitor — over 1/3 of the original trial group experienced a 50% (or greater) increase in rate of weight gain.
In another study, lonafarnib was found to increase the life expectancy of patients by up to a year and a half. Patients in this study also demonstrated a reduction in neurological symptoms like headaches, strokes, and seizures.
Currently, lonafarnib is undergoing trials as a part of a combination therapy with other drugs that treat conditions often related to progeria — such as bone disease, cardiovascular, and chemotherapy drugs.
Eiger Pharmaceuticals plans on submitting a new drug application for lonafarnib to the FDA at some point this year. The company also plans on submitting a marketing authorisation application (MAA) to the European Medicines Agency. If both are approved, lonafarnib will be available for progeria patients throughout the United States and European Union.
Progeria affects only a few hundred people around the world — almost all are children. The availability of a drug that could potentially extend patient’s lives would mean a significant improvement in the quality of life for hundreds of children. What do you think of this exciting development? Share your thoughts with Patient Worthy!
