Sunday, April 14, 2024

Progeria Treatment Zokinvy Receives Japanese Authorization

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The Japan Ministry of Health, Labor and Welfare (MHLW) has approved using Zokinvy, marking an important step in treating progeria, an ultra-rare, fatal pediatric disease characterized by accelerated aging. Zokinvy, a farnesyltransferase inhibitor (FTI), has shown a positive impact on survival rates in children afflicted with this condition.

Clinical tests coordinated by the Progeria Research Foundation (PRF) and Boston Children’s Hospital revealed that Zokinvy can reduce mortality incidence by 72% and increase average survival time by 4.3 years, or 30%. Dr. Monica Kleinman, the principal investigator of the clinical trials at Boston Children’s Hospital, highlighted the significance of this achievement in Progeria research.

Zokinvy Gains Regulatory Approval in Japan, Offering Hope to Progeria Patients

The approval of Zokinvy in Japan follows the drug’s endorsement by the U.S. Food and Drug Administration in November 2020 and the European Medicines Administration in July 2022. These approvals have been hailed as a major advancement in the treatment options available for this condition.

Audrey Gordon, the president and executive director of the Progeria Research Foundation, expressed her optimism for the future, stating that children and young adults with progeria in Japan will now be able to access this life-extending, heart-strengthening treatment through a prescription.

The PRF collaborated with Eiger BioPharmaceuticals to bring Zokinvy to the market. This condition, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is a result of a genetic mutation in the LMNA gene.


Japan Approves Progeria Treatment, Paving the Way for Enhanced Lives

The approval of Zokinvy in Japan adds to the growing global recognition of the drug’s effectiveness and its potential to significantly enhance the quality of life for those affected by this rare condition.

The approval of Zokinvy by the MHLW not only marks a significant milestone in the treatment of this condition but also highlights the transformative potential of scientific advancement in rare disease research. The drug, which has been shown to increase survival rates and time in children with the disease, is now accessible to patients through a prescription, offering hope for an improved quality of life.

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