The European Commission granted marketing authorization for Eiger BioPharmaceuticals’ Zokinvy as the first and only treatment approved in Europe to treat children and young adults with Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PL).
Photo: Thorsten Marquardt, professor of pediatric and adolescent medicine, University of Münster, Germany
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. There are approximately 20 children and young adults with progeria identified across Europe.
“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 Thorsten Marquardt, professor of pediatric and adolescent medicine, University of Münster, Germany. “With less than 5 percent of rare diseases having an approved treatment, the authorization of Zokinvy represents both a significant medical milestone and increased hope for the progeria community.”
The EC authorization follows the positive opinion granted by European Committee for Medicinal Products for Human Use (CHMP) in May 2022 which was based on the positive results of two pivotal clinical trials demonstrating that Zokinvy, an oral disease-modifying agent which targets the cause of progeria, lowered the risk of death in children by 72 percent, and extended life by an average of 4.3 years in children and young adults with HGPS.
The European Commission’s centralized marketing authorization is valid in all 27 EU member states as well as Iceland, Liechtenstein, and Norway. Regulatory review is ongoing by the UK’s Medicine and Healthcare products Regulatory Agency (MHRA) as part of the European Commission Decision Reliance Procedure. Eiger expects to make Zokinvy available following successful completion of reimbursement discussions country-by-country.
HGPS 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.
Without Zokinvy therapy, children with HGPS 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.
Eiger licensed exclusive worldwide rights to Zokinvy from Merck, known as MSD, outside of the United States and Canada. Merck will not receive any milestone payments for the development of Zokinvy for the treatment of progeria and has waived royalty obligations from Eiger for a specified quantity of Zokinvy.
In Europe, Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.
Author: Rare Daily Staff
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