Breaking, exciting news! On November 20, 2020, PRF achieved an important piece of our mission: lonafarnib, the first-ever treatment for Progeria, has been granted FDA approval.
Progeria now joins less than 5% of the rare diseases with an FDA-approved treatment.* Children and young adults with Progeria in the U.S. may now access lonafarnib (now called ‘Zokinvy’) by prescription, instead of through a clinical trial.
This momentous milestone has arrived thanks to 13 steadfast years of research involving four clinical trials, all co-coordinated by PRF, made possible by the courageous children and their families, and funded by you, PRF’s wonderful community of donors.
We’re also grateful to the world-class Progeria research teams at Boston Children’s Hospital, Hasbro Children’s Hospital, Brigham and Women’s Hospital, Brown University, Boston University, and the National Institutes of Health. Pharmaceutical partners that supplied lonafarnib free of charge to PRF-supported clinical trials were crucial as well, including Shering-Plough, Merck ** (known as MSD outside the U.S. and Canada) and Eiger BioPharmaceuticals, for their part in advancing progress in Progeria research to these extraordinary new heights.
Our partners at Eiger are committed to providing continuous access to Zokinvy for patients with Progeria and processing-deficient Progeroid Laminopathies worldwide, and they’ve established services to support access to Zokinvy through Eiger OneCare, as well as through Eiger’s Managed Access Program for patients outside the U.S. The European Medicines Agency (EMA) review of Zokinvy is still ongoing, with a decision on EMA approval expected in the second half of 2021.
THANK YOU ALL for supporting the research that has brought us to this pivotal point. Your continued support also allows us to keep pressing forward toward a cure for these extraordinary children.
What a truly remarkable way to begin the holiday season and end this incredibly challenging year.
Click here for our press release which provides more details on this historic news.
*300 rare diseases that have an FDA-approved treatment (https://www.rarediseases.info.nih.gov/diseases/FDS-orphan-drugs)/7,000 rare diseases for which the molecular basis is known (www.OMIM.org) =4.2%
** PRF would like to acknowledge the critical contributions of the scientists from Schering-Plough / Merck R&D who supported both the evaluation of lonafarnib in preclinical models of HGPS and the clinical studies in progeria patients. This team, led by W. Robert Bishop, John Piwinski, Cecil Pickett, and Catherine Strader, supported pharmacokinetic / pharmacodynamic studies, optimized drug formulation and insured adequate drug supply throughout these studies. Members of this team included: Susan Arbuck, Art Bertelsen, Alan Cooper, Emily Frank, David Harris, Georgianna Harris, Paul Kirschmeier, Ming Liu, Jin-Keon Pai, Robert Patton, Paul Statkevich, Greg Szpunar, Bohdan Yaremko, Paul Zavodny, and Yali Zhu.