Eiger and Partner, AnGes, Receive Approval for Zokinvy® (lonafarnib) for Hutchinson- Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies in Japan
- Clinical trial data demonstrated Zokinvy treatment extended life by an average of 4.3 years in children and young adults with Hutchinson-Gilford progeria
Eiger to receive$500,000 approval milestone payment from AnGes- Zokinvy approved in the
U.S. (2020), 30 European countries (2022), and nowJapan (2024)
"We and our partner, AnGes, are pleased that Zokinvy is now approved in
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.3,4
The approval 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% and extended life by an average of 4.3 years (p<0.0001) in children and young adults with HGPS.2
About Progeria
Collectively known as progeria, Hutchinson-Gilford progeria syndrome and progeroid laminopathies are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients.
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.4,5
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.3
About Zokinvy® (lonafarnib)
Zokinvy is a first-in-class disease-modifying agent that blocks the accumulation of defective progerin and progerin-like proteins which leads to cellular instability and premature aging in children and young adults with progeria. Zokinvy has demonstrated a statistically significant survival benefit in children and young adults with HGPS.1,4
The most commonly reported adverse reactions were gastrointestinal (vomiting, diarrhea, nausea), and most were mild or moderate (Grade 1 or 2) in severity. Many progeria patients have received continuous Zokinvy therapy for more than 10 years.1,2
Zokinvy is FDA approved 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.
For Important Safety Information and prescribing information for Zokinvy in the
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References:
1. Data on file,
2. Summary of Product Characteristics,
3. Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford Progeria Syndrome. 2003 Dec 12
[Updated 2019 Jan 17]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA):
4. Gordon LB, Shappell H, Massaro J, et al. Association of lonafarnib treatment vs no treatment with mortality rate in patients with Hutchinson-Gilford progeria syndrome. JAMA. 2018;319(16):1687-1695. doi:10.1001/jama.2018.3264.
5. Marcelot A, Worman HJ, and
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