Zokinvy is indicated for Hutchinson-Gilford progeria syndrome (HGPS) and processing deficient progeroid laminopathy (PDPL), two ultra-rare paediatric diseases that cause genetic premature ageing diseases that advance mortality in young patients
Zokinvy is approved to treat rare paediatric diseases. (Credit: Myriams-Fotos from Pixabay)
US-based biopharmaceutical company Eiger BioPharmaceuticals and its partner AnGes have received Japan’s Ministry of Health, Labour, and Welfare (MHLW) for Zokinvy (lonafarnib).
Zokinvy is a prescription treatment indicated for Hutchinson-Gilford progeria syndrome (HGPS) and processing deficient progeroid laminopathy (PDPL).
It works by blocking the accumulation of progerin and progerin-like proteins which may lead to cellular instability and premature ageing in children and young adults with progeria.
Collectively known as progeria, HGPS and PL are ultra-rare and serious paediatric diseases that cause genetic premature ageing diseases that advance mortality in young patients.
Eiger CEO David Apelian said: “We and our partner, AnGes, are pleased that Zokinvy is now approved in Japan for patients living with progeria, an ultra-rare and fatal pediatric disease that can result in premature death.
“We would like to thank the Progeria Research Foundation for their continued support of the regulatory submission as well as the patients and their families.”
The regulatory approval in Japan was supported by the positive results from two clinical trials, which showed that Zokinvy targets the cause of progeria.
In the studies, Zokinvy showed a statistically significant survival benefit in children and adults.
The treatment lowered the risk of death in children by 72% and extended life by an average of 4.3 years in children and young adults with HGPS.
The most commonly reported adverse reactions include vomiting, diarrhoea, and nausea, most of which were mild or moderate in severity.
Zokinvy was previously approved in the US, to treat patients aged 12 months and above, with a genetically confirmed diagnosis of HGPS or PL.
In May 2022, Eiger and AnGes signed an exclusive distribution agreement for Zokinvy (Lonafarnib) to treat HGPS and PDPL in Japan.
The European Commission (EC) authorised Zokinvy to treat HGPS and PDPL, in July 2022.
In March last year, Japan’s health ministry granted Zokinvy as an orphan drug designation.