Lonafarnib
After the 2003 discovery of the Progeria gene, researchers identified lonafarnib as a promising treatment candidate. In a PRF-funded, landmark trial that began in 2007, 28 children from 16 countries — 75% of then-known cases worldwide — traveled to Boston Children's Hospital over a period of two years for comprehensive testing and twice-daily oral lonafarnib. In 2020, lonafarnib became the first FDA approved drug for Progeria under the brand name Zokinvy®.
Lonafarnib works by blocking the farnesyl group from attaching to progerin, the mutant protein that causes Progeria's accelerated aging. Without that attachment, progerin cannot disrupt normal cell function.
Every child enrolled in the clinical trial experienced measurable improvement — most critically in the cardiovascular system, the primary cause of death in Progeria. Bone density increased, weight gain improved, and arterial stiffness was reduced. A 2012 publication in PNAS documented these cardiovascular and structural gains across the full trial cohort. A landmark 2018 study in JAMA then confirmed what no treatment had ever shown before: lonafarnib extended survival by at least 1.6 years.
Every child previously enrolled in a PRF trial continues to receive lonafarnib — at no cost to families — through PRF's monotherapy extension or Managed Access Program.
1.6+
Years extended survival confirmed in JAMA, 2018
100%
Of children showed improvement in at least one clinical measure
28
Children enrolled from 16 countries in the first lonafarnib trial
2020
FDA accelerated approval as Zokinvy
The lonafarnib managed access program (MAP) is managed by Sentynl Therapeutics. MAP allows eligible children and young adults with Progeria to obtain the drug lonafarnib through their local physicians in countries that allow the MAP to be offered.
PDF program details are available in multiple languages below:
Shelby works with newly identified families to introduce them to PRF programs. She is the main point of contact for families interested in contributing to The Progeria Research Foundation’s Cell & Tissue Bank or participating in the Diagnostic Testing Program and Medical & Research Database Program.
Shelby keeps data for the Natural History Study and assists the Medical Director with data-keeping for additional internal research projects. She also helps interested families and their physicians to enroll in Sentynl’s Managed Access Program for lonafarnib.
Please contact Shelby at [email protected].
