European approval for Zokinvy for Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies.- Eiger BioPharmceuticals
Eiger BioPharmaceuticals Inc. announced the European Commission (EC) has granted marketing authorization for Zokinvy (lonafarnib), a first-in-class breakthrough therapy for children and young adults with Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PL).
Collectively known as progeria, HGPS and PL are devastating ultra-rare, and fatal pediatric diseases that cause dramatically accelerated aging and premature death. The main cause of death is heart attack or stroke due to severe hardening of the arteries. Without Zokinvy treatment, children with HGPS die at an average age of 14.5 years.
"Until now, no medication has been approved in Europe for people living with this debilitating condition. For the first time we have a therapeutic option with Zokinvy which has been proven to meaningfully extend the average life span of children with HGPS by nearly one third," said Professor Thorsten Marquardt, Paediatric and Adolescent Medicine, University of Münster, Germany. "With less than 5% of rare diseases having an approved treatment, the authorization of Zokinvy represents both a significant medical milestone and increased hope for the progeria community."
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