Rare Daily Staff
Eiger BioPharmaceuticals announced the commercial launch and availability of Zokinvy in the United States, the first and only approved treatment for the rare premature aging disease progeria.
Zokinvy was approved by the U.S. Food and Drug Administration in November 2020 to reduce the risk of death in in adult and pediatric patients 12 months of age and older with Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and for the treatment of processing-deficient progeroid laminopathies.
Progeria and progeroid Laminopathies are separate and distinct ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients. Progeria is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin. Progeroid Laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the LMNA and/or Zmpste24 genes yielding farnesylated proteins that are distinct from progerin. While non–progerin producing, these genetic mutations result in disease manifestations with phenotypes that overlap with, but are distinct from, progeria.
Children with progeria commonly die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), by an average age of 14.5 years. Disease manifestations include severe failure to thrive, scleroderma–like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes. It is estimated that there are 400 children worldwide with progeria and 200 children with progeroid laminopathies. Of these patients, the Progeria Research Foundation has identified approximately 180 children and young adults, including approximately 20 in the United States and 23 in Europe.
Zokinvy (lonafarnib) blocks the accumulation of defective, farnesylated proteins which form tight associations with the nuclear envelope, leading to cellular instability and the process of premature aging in children and young adults with progeria and processing-deficient progeroid laminopathies.
Eiger licensed exclusive worldwide rights to lonafarnib from Merck, which will not receive any milestone payments for the development of lonafarnib for the treatment of progeria and has waived royalty obligations from Eiger for a specified quantity of lonafarnib. Zokinvy was developed in collaboration with the Progeria Research Foundation.
The company has established Eiger OneCare to help patients with progeria and progeroid laminophathies. The program includes personalized support by specialized care managers, reimbursement experts, co-pay assistance for eligible patients and other patient support services designed to assist patients seeking access to Zokinvy.
“Eiger sought regulatory approval of Zokinvy to ensure continued access to the only drug proven to reduce the risk of death in patients with progeria,” said Eldon Mayer, chief commercial officer of Eiger.
Most of the identified Zokinvy-eligible patients in the United States have received Zokinvy through Eiger’s global expanded access program and are now enrolled in Eiger OneCare, which the company says will facilitate a smooth transition to Zokinvy commercial supply.
Photo: Eldon Mayer, chief commercial officer of Eiger.
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