The Progeria Research Foundation (PRF) celebrates a significant milestone as Zokinvy™ (lonafarnib) receives approval from the Japanese Ministry of Health, Labor and Welfare (MHLW) for treating Progeria and processing-deficient Progeroid Laminopathies (PL). Progeria, an ultra-rare, fatal pediatric 'rapid-aging' disease, has garnered attention due to the life-extending potential of Zokinvy.

PRF, a pioneering force in rare disease research, has been at the forefront of Zokinvy clinical trial research since 2007. In collaboration with Eiger BioPharmaceuticals (Eiger), PRF embarked on a partnership in 2018 to bring this groundbreaking drug to market. Zokinvy received approval from the US Food and Drug Administration (FDA) in November 2020 and the European Medicines Administration (EMA) in July 2022.

Zokinvy, a farnesyltransferase inhibitor (FTI), has demonstrated a survival benefit in children with Progeria. Clinical trial data, including information from the PRF International Patient Registry and trials coordinated by PRF and Boston Children's Hospital (BCH), reveals a 72 percent reduction in mortality incidence and a 30 percent increase in average survival time (4.3 years) for patients with Progeria. Without Zokinvy, children with Progeria typically succumb to heart disease at an average age of 14.5 years.

"We're thrilled by this wonderful news," said Audrey Gordon, President and Executive Director of The Progeria Research Foundation. "On the heels of the 2020 FDA approval and the 2022 EMA approval for Zokinvy, now children and young adults with Progeria and PL in Japan will be able to access this life-extending, heart-strengthening treatment through the convenience of a prescription."

Leslie Gordon, MD, PhD, PRF Medical Director, and Zokinvy Clinical Trial Investigator, expressed gratitude to the children, families, and research teams involved, stating, "After more than a decade of Zokinvy research funded by PRF and co-conducted by PRF and BCH, Zokinvy's authorization in Japan is a win for the worldwide Progeria community."

PRF, in its 24-year journey, has played a crucial role in Progeria research, from the 2003 gene discovery to groundbreaking initiatives in RNA therapeutics and Genetic Base Editing. The foundation's patient-focused programs include the Progeria International Patient Registry, Medical & Research Database, Cell & Tissue Bank, Diagnostics Testing Program, and a Clinical Care Handbook for families and physicians.

Dr. Monica Kleinman, Principal Investigator for the Progeria clinical trials at Boston Children's Hospital, remarked, "Being a part of this achievement for these children is not only professionally rewarding but also a testament to the transformative potential of scientific advancement in Progeria research."